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Latest FDA New Drug Application News

Kura Oncology and Kyowa Kirin Announce Submission of New Drug Application for Ziftomenib to FDA

April 8
Last Trade: 5.62 -0.14 -2.43

SAN DIEGO and TOKYO, April 08, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”), and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”), today announced Kura submitted a New Drug Application (NDA) for ziftomenib, a highly selective, once-daily, oral, investigational menin inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM 1) mutation to...Read more


ALX Oncology Receives IND Clearance from U.S. FDA for ALX2004, a Novel EGFR-targeted Antibody-drug Conjugate

April 7
Last Trade: 0.51 -0.03 -5.47

ALX2004 is a potential best- and first-in-class antibody-drug conjugate (ADC) for the treatment of EGFR-expressing solid tumors that was created from ALX Oncology’s proprietary linker-payload platform ALX2004, the company’s first ADC, was fully designed and developed in-house by ALX Oncology scientists Company expects to initiate Phase 1 clinical trials of ALX2004 in mid-2025, with initial safety data available in 1H 2026 SOUTH SAN...Read more


CureVac Receives U.S. FDA IND Clearance to Initiate Phase 1 Clinical Trial for Novel mRNA-Based Precision Immunotherapy in Squamous Non-Small Cell Lung Cancer

April 7
Last Trade: 2.72 0.07 2.64

Significant Regulatory Milestone: U.S. FDA cleared IND application for CVHNLC, CureVac's investigational therapy targeting squamous non-small cell lung cancer Proprietary Epitopes: CVHNLC encodes novel tumor epitopes identified through proprietary whole genome-based discovery platform Enhanced Combination Therapy: CVHNLC to be tested in combination with pembrolizumab, aiming to amplify targeted anti-tumor immune...Read more


Apellis Pharmaceuticals Announces FDA Acceptance and Priority Review of the Supplemental New Drug Application for EMPAVELI® (pegcetacoplan) for C3G and Primary IC-MPGN

April 1
Last Trade: 18.70 -1.10 -5.56

PDUFA target action date is July 28, 2025 WALTHAM, Mass., April 01, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the supplemental New Drug Application (sNDA) for EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis...Read more


Ascendis Pharma Submits U.S. NDA for TransCon CNP (Navepegritide) for the Treatment of Children with Achondroplasia

March 31
Last Trade: 137.41 -2.73 -1.95

Data demonstrated multiple clinical benefits beyond linear growth NDA supported by data from three randomized, double-blind, placebo-controlled clinical trials in children with achondroplasia, with up to three years of open-label extension data MAA in EU on track for submission during Q3 2025 COPENHAGEN, Denmark, March 31, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that it has submitted its New...Read more


Vanda Pharmaceuticals Announces the Submission of an NDA to the FDA for Bysanti™ for the Treatments of Acute Bipolar I Disorder and Schizophrenia

March 31
Last Trade: 4.14 -0.05 -1.19

WASHINGTON, March 31, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (NASDAQ: VNDA) today announced that a New Drug Application (NDA) was submitted to the U.S. Food and Drug Administration (FDA) requesting marketing approval of Bysanti™ (milsaperidone) for the treatments of acute bipolar I disorder and schizophrenia. The NDA is supported by several clinical studies assessing the efficacy and safety of Bysanti™. Bysanti™ is a new...Read more


Liquidia Announces FDA Acceptance of New Drug Application Resubmission for YUTREPIA™ (treprostinil) Inhalation Powder

March 28
Last Trade: 11.99 -0.44 -3.54

MORRISVILLE, N.C., March 28, 2025 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA), a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary disease, today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) resubmission for YUTREPIA™ (treprostinil) inhalation powder to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension...Read more


Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) by the United States (U.S.) Food and Drug Administration

March 27
Last Trade: 14.41 -0.89 -5.82

U.S. IND Represents Regulatory Clearance For BEAM-302 Spanning Six Countries and Second Open IND for a Beam In Vivo Base Editing Program in the U.S. CAMBRIDGE, Mass., March 27, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has cleared the...Read more


Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-102, an Investigational Gene Editing Medicine Designed to Durably Lower Cholesterol After a Single Dose

March 24
Last Trade: 2.97 -0.37 -11.08

BOSTON, March 24, 2025 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage company developing a new class of genetic medicines for cardiovascular disease, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for VERVE-102 for the treatment of patients living with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD)....Read more


Telix Pharmaceuticals: FDA Approves New Prostate Cancer Imaging Agent Gozellix®

March 21
Last Trade: 14.31 0.06 0.42

MELBOURNE, Australia and INDIANAPOLIS, March 21, 2025 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Nasdaq: TLX, Telix, the Company) today announces that the United States (U.S.) Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for Gozellix® (TLX007-CDx, kit for the preparation of gallium-68 (68Ga) gozetotide injection), Telix’s next-generation PSMA-PET imaging1 agent for prostate...Read more


Scienture announces the U.S. FDA Approval of its NDA for SCN-102, to be launched as ArbliTM, (losartan potassium) Oral Suspension, 10mg/mL. The global market for losartan potassium was approximately $1.5 billion in sales in 2024.

March 18
Last Trade: 0.85 -0.08 -8.60

TAMPA, FL, March 18, 2025 (GLOBE NEWSWIRE) -- – SCIENTURE HOLDINGS, INC. NASDAQ: SCNX), a holding company for existing and planned pharmaceutical operating companies focused on providing enhanced value to patients, physicians and caregivers through developing, bringing to market, and distributing novel specialty products to satisfy unmet market needs, is pleased to announce the U.S. Food and Drug Administration (FDA) has approved...Read more


Xeris Biopharma Announces Approval of Supplemental New Drug Application (sNDA) of Gvoke VialDx™ (glucagon) for Use as a Diagnostic Aid

March 17
Last Trade: 4.13 -0.28 -6.35

First concentrated, ready-to-dilute liquid glucagon available for growing procedural gastroenterology market American Regent to commercialize GVOKE VialDx™ Availability is expected in the third quarter of 2025 CHICAGO / Mar 17, 2025 / Business Wire / Xeris Biopharma Holdings, Inc. (Nasdaq: XERS), a growth-oriented biopharmaceutical company committed to improving patient lives by developing and commercializing innovative products...Read more


Precision BioSciences Announces Clearance of Investigational New Drug Application by the U.S. FDA for First-in-Class PBGENE-HBV Designed to Eliminate Root Cause of Chronic Hepatitis B

March 17
Last Trade: 4.62 0.22 5.00

PBGENE-HBV is the first-ever investigational in vivo gene editing therapy cleared to enter clinical trials for the treatment of chronic hepatitis B in the United States (U.S.)  IND clearance represents a significant regulatory milestone for PBGENE-HBV  Company to expand its Phase 1 ELIMINATE-B study to the U.S., joining world-class clinical sites in Moldova, Hong Kong, and New Zealand where strong clinical execution is...Read more


Travere Therapeutics Submits sNDA to FDA for Approval of FILSPARI® (sparsentan) for the Treatment of FSGS

March 17
Last Trade: 14.15 -1.02 -6.72

sNDA submission based on results from Phase 3 DUPLEX and Phase 2 DUET studies of FILSPARI in FSGS If approved, FILSPARI could become the first and only FDA-approved treatment for FSGS, a rare kidney condition and a leading cause of kidney failure Additionally, the FDA notified the Company that REMS monitoring for embryo-fetal toxicity is no longer necessary; the Company plans to submit an amendment to the REMS sNDA currently under...Read more


PDS Biotechnology Announces FDA Clearance of IND Application for Combination of Versamune® MUC1 and PDS01ADC to Treat Metastatic Colorectal Cancer

March 13
Last Trade: 0.94 -0.03 -2.93

Phase 1/2 trial evaluating the proprietary combination to be run under CRADA with the National Cancer Institute Second Versamune® platform candidate targets MUC1-positive solid tumors U.S. Patent #12,201,685 covering methods of using combinations of the Versamune® platform and various immunocytokines, including PDS01ADC recently issued PRINCETON, N.J., March 13, 2025 (GLOBE NEWSWIRE) -- PDS Biotechnology Corporation (Nasdaq: PDSB)...Read more


Sagimet Biosciences Announces Clearance of IND for FASN Inhibitor TVB-3567, to be Developed for the Treatment of Acne

March 11
Last Trade: 1.90 -0.04 -2.06

TVB-3567 is the Company’s second fatty acid synthase (FASN) inhibitor First-in-human Phase 1 trial initiation planned in 2025 SAN MATEO, Calif., March 11, 2025 (GLOBE NEWSWIRE) -- Sagimet Biosciences Inc. (Sagimet, Nasdaq: SGMT), a clinical-stage biopharmaceutical company developing novel therapeutics targeting dysfunctional metabolic and fibrotic pathways, today announced the clearance of its Investigational New Drug (IND)...Read more


scPharmaceuticals Announces FDA Approval of Supplemental New Drug Application Expanding the FUROSCIX® Indication to Include the Treatment of Edema in Patients with Chronic Kidney Disease

March 6
Last Trade: 2.15 -0.18 -7.73

FDA approves scPharmaceuticals’ supplemental New Drug Application to expand the FUROSCIX indication to include the treatment of edema in adult patients with chronic kidney disease, including nephrotic syndrome FUROSCIX is expected to be available for chronic kidney disease patients in April 2025 BURLINGTON, Mass., March 06, 2025 (GLOBE NEWSWIRE) -- scPharmaceuticals Inc. (Nasdaq: SCPH) (the “Company”), a pharmaceutical company...Read more


Tiziana Life Sciences Files Investigational New Drug Application with FDA for ALS Phase 2 Clinical Trial

March 4
Last Trade: 0.80 -0.10 -10.80

NEW YORK, March 04, 2025 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3 monoclonal antibody, today announced the submission of its Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a phase 2...Read more


FDA Files Corcept’s New Drug Application for Relacorilant as Treatment for Patients With Hypercortisolism

March 3
Last Trade: 70.49 -4.16 -5.57

FDA assigns a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2025 REDWOOD CITY, Calif. / Mar 03, 2025 / Business Wire / Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, today announced that the U.S....Read more


Sernova Biotherapeutics Receives FDA Clearance for IND Application for its Cell Pouch Bio-hybrid Organ with Autograft Thyroid Cells in Patients with Hypothyroidism

March 3
Last Trade: 0.18 0.005 2.94

LONDON, Ontario and BOSTON, March 03, 2025 (GLOBE NEWSWIRE) -- Sernova Biotherapeutics, (TSX: SVA) (OTCQB: SEOVF) (FSE/XETRA: PSH), a leading regenerative medicine company focused on developing it’s Cell Pouch bio-hybrid organ as a functional cure for type 1 diabetes, today announced that the U.S. Food and Drug Administration (FDA) has cleared it’s Investigational New Drug (IND) application to evaluate the Company’s Cell Pouch with...Read more


PharmaTher Provides Complete Response Amendment for US FDA New Drug Application for Ketamine

March 3
Last Trade: 0.18 0.005 2.86

Expecting FDA approval of Ketamine in Q2-2025 Solving the shortage problem of Ketamine and unlocking its pharmaceutical potential Toronto, Ontario--(Newsfile Corp. - March 3, 2025) - PharmaTher Holdings Ltd. (OTCQB: PHRRF) (CSE: PHRM) (the "Company" or "PharmaTher"), a specialty pharmaceutical company, is pleased to announce today that the Company has provided its resubmission to the complete response amendment for its U.S. Food and...Read more


BioRestorative Therapies Announces FDA Clearance of IND for Phase 2 Trial of BRTX-100 in Chronic Cervical Discogenic Pain

February 27
Last Trade: 1.76 0.06 3.53

BRTX-100 is the first and only stem cell product candidate cleared to be evaluated in the cervical degenerative disc disease setting IND clearance expands Company’s advanced clinical pipeline for BRTX-100 treatment of both chronic lower back and neck pain Company to host webcasted conference call today at 10:30am EST MELVILLE, N.Y., Feb. 27, 2025 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (“BioRestorative”, “BRTX” or the...Read more


Arcutis Biotherapeutics: U.S. FDA Accepts Supplemental New Drug Application for Arcutis’ ZORYVE® (roflumilast) Cream 0.05% for the Treatment of Children Aged 2 to 5 with Mild to Moderate Atopic Dermatitis 

February 26
Last Trade: 13.30 -1.00 -6.99

U.S. Food and Drug Administration (FDA) has set a Prescription Drug User Fee Act (PDUFA) target action date of October 13, 2025  Application supported by positive efficacy and safety data from pivotal Phase 3 trial, pivotal long-term extension study, and Phase 1 pharmacokinetic study  If approved, ZORYVE cream 0.05% will be a new topical therapy option for approximately 1.8 million children aged 2 to 5 with atopic dermatitis...Read more


Teva Pharmaceutical and Medincell Announce FDA Acceptance of Supplemental New Drug Application for UZEDY(R) (risperidone) Extended-Release Injectable Suspension as a Treatment for Patients with Bipolar I Disorder

February 25
Last Trade: 13.59 -0.35 -2.51

UZEDY is currently approved in the US as a subcutaneous long-acting injectable (LAI) for use every one or two months for the treatment of schizophrenia in adults1 LAI treatment options may help address unmet needs of people living with bipolar I disorder (BP-I) BP-I filing acceptance for UZEDY represents Teva’s commitment to pursuing new advances in neuroscience PARSIPPANY, N.J. and TEL AVIV, Israel and PARIS, Feb. 25, 2025 (GLOBE...Read more


Glaukos Announces FDA Acceptance of NDA Submission for Epioxa™

February 24
Last Trade: 82.27 -3.80 -4.42

PDUFA Date Set for October 20, 2025 ALISO VIEJO, Calif. / Feb 24, 2025 / Business Wire / Glaukos Corporation (NYSE: GKOS), an ophthalmic pharmaceutical and medical technology company focused on novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases, today announced it has received the “Day 74” notification from the U.S. Food and Drug Administration (FDA) acknowledging the previously submitted New Drug...Read more


Amneal Pharmaceuticals Launches Mesalamine and Receives U.S. FDA Approval for Lenalidomide

February 19
Last Trade: 6.99 -0.22 -3.05

Mesalamine 800 mg delayed-release tablets, referencing ASACOL HD® Lenalidomide capsules, referencing REVLIMID® BRIDGEWATER, N.J. / Feb 19, 2025 / Business Wire / Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”), a global biopharmaceutical company, today announced the launch of mesalamine 800 mg delayed-release tablets, an aminosalicylate indicated for the treatment of moderately active ulcerative colitis in...Read more


PTC Therapeutics Announces FDA Acceptance and Priority Review for Vatiquinone NDA for the Treatment of Children and Adults with Friedreich's Ataxia

February 19
Last Trade: 39.77 -3.05 -7.12

PDUFA target action date of Aug. 19, 2025  WARREN, N.J., Feb. 19, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia (FA). The application has been granted Priority Review and assigned a Prescription Drug User...Read more


U.S. FDA Accepts Gilead’s New Drug Applications for Twice-Yearly Lenacapavir for HIV Prevention Under Priority Review

February 18
Last Trade: 103.19 -2.33 -2.21

If Approved, Lenacapavir Would Be the First and Only Twice-Yearly HIV Prevention Choice  FDA to Review Applications Under Priority Review, with a PDUFA Date of June 19, 2025  Gilead Also Recently Submitted Applications for Lenacapavir for PrEP to the European Medicines Agency That Will Be Reviewed Under Accelerated Assessment Review Timeline  FOSTER CITY, Calif. / Feb 18, 2025 / Business Wire / Gilead Sciences, Inc....Read more


Chimerix Announces FDA Acceptance and Priority Review of New Drug Application for Dordaviprone as Treatment for Recurrent H3 K27M-Mutant Diffuse Glioma

February 18
Last Trade: 8.51 -0.01 -0.12

Potential First Approval for Patients with Recurrent H3 K27M-mutant Diffuse Glioma PDUFA Target Action Date of August 18, 2025 No Advisory Committee Meeting Currently Planned to Discuss Application DURHAM, N.C., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ: CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today...Read more


Biohaven Announces FDA Acceptance and Priority Review of Troriluzole New Drug Application for the Treatment of Spinocerebellar Ataxia

February 11
Last Trade: 16.54 -1.37 -7.65

Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. Troriluzole demonstrated a 50-70% slowing of SCA disease progression on the primary and secondary outcome measures at the 3-year endpoint in a real-world evidence (RWE) study. Troriluzole has a well-established safety profile and if approved, would be the first and only FDA-approved treatment for SCA; subject to...Read more


FDA Grants Priority Review to Insmed's Brensocatib for Treatment of Bronchiectasis with PDUFA Target Action Date Set for August 12, 2025

February 6
Last Trade: 64.97 -3.12 -4.58

Brensocatib Would be the First and Only Available Treatment for Bronchiectasis and First DPP1 Inhibitor, if Approved BRIDGEWATER, N.J., Feb. 6, 2025 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has...Read more


Sernova Biotherapeutics Submits IND Application for its Cell Pouch Bio-Hybrid Organ with Autograft Thyroid Cells in Patients with Hypothyroidism

February 3
Last Trade: 0.18 0.005 2.94

LONDON, Ontario and BOSTON, Feb. 03, 2025 (GLOBE NEWSWIRE) -- Sernova Biotherapeutics, (TSX: SVA) (OTCQB: SEOVF) (FSE/XETRA: PSH), a leading regenerative medicine company focused on developing its Cell Pouch bio-hybrid organ as a functional cure for type 1 diabetes, today announced the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to evaluate the Company’s Cell Pouch...Read more


HCW Biologics Granted FDA Clearance to Evaluate One of the Company’s Lead Product Candidates in a First-In-Human Phase 1 Clinical Trial

February 3
Last Trade: 0.23 0.0079 3.56

MIRAMAR, Fla., Feb. 03, 2025 (GLOBE NEWSWIRE) -- HCW Biologics Inc. (“HCWB” or the “Company”) (NASDAQ: HCWB), a U.S.-based clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies to extend healthspan by targeting the link between chronic inflammation and disease, today announced that it has received clearance of its Investigational New Drug Application (“IND”) from the U.S. Food and Drug...Read more


United Therapeutics Announces FDA Clearance of its Investigational New Drug Application for the UKidney Xenotransplantation Clinical Trial

February 3
Last Trade: 279.69 -12.03 -4.12

First-ever human clinical trial of a xeno-organ intended to allow for potential registration through the submission of a Biologics License Application to the U.S. FDA First xenotransplant is expected to occur around mid-year 2025 SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C. / Feb 03, 2025 / Business Wire / United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that the U.S. Food and Drug...Read more


Ironwood Pharmaceuticals Initiates Apraglutide NDA Submission, Highlights Compelling New Data and Updates Corporate Structure to Position for the Future

January 29
Last Trade: 1.11 -0.16 -12.60

Data from open label extension study of apraglutide demonstrate further increase in patients achieving enteral autonomy  Initiated rolling NDA submission, now to include long-term extension data given continued clinical improvement over time, with submission completion expected in Q3 2025  Organizational restructuring positions the Company to realize the potential of apraglutide in SBS and long-term success in GI and rare...Read more


Allogene Therapeutics Secures U.S. FDA IND Clearance for ALLO-329, Advancing its Next-Generation Allogeneic CAR T into Autoimmune Diseases

January 28
Last Trade: 1.36 -0.07 -4.90

Dual-Targeted CD19/CD70 Allogeneic CAR T: Best-in-Class Design to Enhance Therapeutic Benefit and Expand Treatment Potential Across a Range of Autoimmune Disease Indications Innovative Dagger® Technology: Empowers ALLO-329 to Overcome Rejection, Potentially Reducing or Eliminating Reliance on Traditional Lymphodepletion Phase 1 RESOLUTION Rheumatology Basket Trial: Initiation Planned for Mid-2025, Targeting Proof-of-Concept to...Read more


FDA Grants Priority Review to Merck’s Application for WELIREG® (belzutifan) for the Treatment of Patients With Advanced Pheochromocytoma and Paraganglioma (PPGL)

January 27
Last Trade: 78.95 -2.23 -2.75

Acceptance based on objective response rate data from the Phase 2 LITESPARK-015 trial evaluating WELIREG in certain patients with advanced PPGL If approved, WELIREG would be the only available therapy in the U.S. for eligible patients with advanced PPGL RAHWAY, N.J. / Jan 27, 2025 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the U.S. Food and Drug Administration (FDA) has...Read more


Ocugen Announces Investigational New Drug Application in Effect After Review by FDA to Initiate Phase 1 Clinical Trial Evaluating First-in-Class OCU500 Inhaled Vaccine Candidate for COVID-19

January 27
Last Trade: 0.59 -0.06 -8.60

OCU500 will be administered via inhalation and as a nasal spray COVID-19 remains a substantial public health threat in the U.S. and around the world Phase 1 clinical trial is anticipated to start in 2Q 2025 MALVERN, Pa., Jan. 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics,...Read more


Biogen: FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA

January 23
Last Trade: 114.05 -5.14 -4.31

CAMBRIDGE, Mass., Jan. 23, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA). The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg...Read more


Johnson & Johnson: SPRAVATO® (esketamine) approved in the U.S. as the first and only monotherapy for adults with treatment-resistant depression

January 21
Last Trade: 150.00 -0.62 -0.41

Following U.S. FDA Priority Review, approval is based on data demonstrating SPRAVATO® alone met its primary endpoint at 4 weeks and led to rapid and superior improvement in depressive symptoms compared to placebo as early as 24 hours1   SPRAVATO® alone showed a rapid and superior improvement vs. placebo in the Montgomery-Asberg Depression Rating Scale (MADRS) total score, with numerical improvements across all 10 MADRS items...Read more


Scilex Announces that It Will Be Filing Today of a Supplemental New Drug Application with the FDA for ELYXYB® in Acute Pain Indication

January 21
Last Trade: 0.25 -0.0015 -0.60

ELYXYB®, a rapid onset and ready-to-use formulation of Celecoxib, delivers a first line non-opioid therapeutic alternative to habit-forming opioids and acetaminophen, the leading cause of acute liver failure in the U.S.(1) DelveInsight estimates there were approximately 100 million cases of acute pain in the United States and that the total acute pain market in the U.S. was approximately $3 billion in 2021.(2) Approximately 40 million...Read more


Arrowhead Pharmaceuticals Announces Acceptance of New Drug Application by U.S. FDA of Plozasiran for the Treatment of Familial Chylomicronemia Syndrome

January 17
Last Trade: 9.99 -0.26 -2.54

The New Drug Application is based on positive results from the Phase 3 PALISADE study People living with familial chylomicronemia syndrome have extremely high triglyceride levels and a substantially higher risk of developing acute pancreatitis and associated long-term complications, including poor quality of life PASADENA, Calif. / Jan 17, 2025 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the...Read more


Johnson & Johnson: New Drug Application initiated with U.S. FDA for TAR-200, the first and only intravesical drug releasing system for patients with BCG-unresponsive high-risk non-muscle-invasive bladder cancer

January 15
Last Trade: 150.00 -0.62 -0.41

Application accepted for U.S. FDA Real-Time Oncology Review (RTOR) based on Phase 2b SunRISe-1 study showing highest single-agent complete response rate of 83.5 percent1 RARITAN, N.J., Jan. 15, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced it has initiated the submission of an original New Drug Application with the U.S. Food and Drug Administration (FDA) for TAR-200 for the treatment of patients with Bacillus...Read more


Tyra Biosciences Receives IND Clearance from FDA to Proceed with Phase 2 Study of TYRA-300 in Non-Muscle Invasive Bladder Cancer (SURF302)

January 10
Last Trade: 7.06 -0.32 -4.34

TYRA appoints urologic oncologist, Erik Goluboff, M.D., as SVP, Clinical Development to lead NMIBC First patient expected to be dosed in SURF302 in Q2 2025 CARLSBAD, Calif., Jan. 10, 2025 /PRNewswire/ -- Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, announced...Read more


FDA Accepts Agios Pharmaceuticals’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia

January 8
Last Trade: 24.53 -1.37 -5.29

CAMBRIDGE, Mass., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) for the treatment of adult patients with non-transfusion-dependent and...Read more


Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich’s Ataxia

January 7
Last Trade: 2.67 -0.27 -9.18

SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich’s ataxia  Dual route of administration enables direct delivery of AAV-based gene therapy to the cerebellum and heart to potentially address the most significant symptoms of the disease  Phase 1b clinical trial initiation expected in 2H 2025  Company to hold a conference call tomorrow,...Read more


Fortress Biotech and Cyprium Therapeutics Announce U.S. FDA Acceptance and Priority Review of NDA for CUTX-101 for Treatment of Menkes Disease

January 6
Last Trade: 1.43 -0.04 -2.72

Priority review granted for CUTX-101 with PDUFA target action date set for June 30, 2025 Cyprium is eligible to receive royalties and up to $129 million in aggregate development and sales milestones Cyprium also retains ownership over any Priority Review Voucher that may be issued at NDA approval MIAMI, Jan. 06, 2025 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) and its majority-owned subsidiary, Cyprium...Read more


Fortress Biotech: Sentynl Therapeutics Announces U.S. FDA Acceptance and Priority Review of New Drug Application for CUTX-101 (Copper Histidinate) Product Candidate for Treatment of Menkes Disease

January 6
Last Trade: 1.43 -0.04 -2.72

Six-month priority review granted for CUTX-101 copper histidinate with PDUFA target action date set for June 30, 2025 CUTX-101 has potential to be the first FDA-approved treatment for Menkes disease, a rare and fatal pediatric disease SOLANA BEACH, Calif. and AHMEDABAD, India and MIAMI, Jan. 6, 2025 /PRNewswire/ -- Sentynl Therapeutics, Inc. ("Sentynl"), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences,...Read more


ANI Pharmaceuticals Announces the FDA Approval and Launch of Prucalopride Tablets with 180-Day CGT Exclusivity

January 2
Last Trade: 63.05 -3.94 -5.88

PRINCETON, N.J., Jan. 02, 2025 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that following final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA), the Company has launched Prucalopride Tablets. ANI’s Prucalopride Tablets is the generic version of the reference listed drug (RLD) Motegrity®. "We are delighted to build momentum...Read more


Corcept Therapeutics Submits New Drug Application for Relacorilant as a Treatment for Patients With Hypercortisolism

December 30
Last Trade: 70.49 -4.16 -5.57

REDWOOD CITY, Calif. / Dec 30, 2024 / Business Wire / Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, has submitted a new drug application (NDA) to the U.S. Food and Drug Administration for its proprietary, selective cortisol...Read more


Chimerix Submits Dordaviprone New Drug Application for Accelerated Approval to U.S. FDA for Patients with Recurrent H3 K27M-Mutant Diffuse Glioma

December 30
Last Trade: 8.51 -0.01 -0.12

Secures Access of Up To $30 Million to Support Potential Launch Through Silicon Valley Bank Credit Facility DURHAM, N.C., Dec. 30, 2024 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ: CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today confirms that the Company has submitted a New Drug Application (NDA) with the U.S. Food and Drug...Read more


Verastem Oncology Announces FDA Acceptance and Priority Review of New Drug Application for Avutometinib in Combination with Defactinib for the Treatment of Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer

December 30
Last Trade: 4.65 -0.46 -9.02

PDUFA target action date is June 30, 2025 If approved, avutometinib in combination with defactinib would be the first-ever FDA-approved treatment specifically for adults with recurrent KRAS mutant LGSOC BOSTON / Dec 30, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has...Read more


NRx Pharmaceuticals Files Initial Section of U.S. New Drug Application to the FDA for NRX-100 (IV Ketamine) for the Treatment of Suicidal Depression

December 30
Last Trade: 1.71 -0.10 -5.52

Aiming to be the first FDA-approved medication to treat suicidal depression Designed to help address the needs of the more than 13 million Americans who seriously consider suicide each year (CDC) Completion of NDA filing expected in the first quarter of 2025 Company to participate in 1x1 meetings in San Francisco during the Annual J.P. Morgan Healthcare Conference on January 13-16, 2025, in San Francisco, CA.  To schedule...Read more


U.S. Food and Drug Administration Accepts for Priority Review Nuvation Bio’s New Drug Application for Taletrectinib for the Treatment of Advanced ROS1-positive Non-Small Cell Lung Cancer

December 23
Last Trade: 1.67 -0.13 -7.22

New Drug Application (NDA) is based on pooled data from the pivotal Phase 2 TRUST-I and TRUST-II studies of taletrectinib that demonstrated durable responses and prolonged progression-free survival in patients with advanced ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC) If approved, taletrectinib represents a potential best-in-class treatment option for patients with advanced ROS1+ NSCLC U.S. Food and Drug Administration (FDA)...Read more


Glaukos Submits New Drug Application to U.S. FDA for Epioxa™

December 23
Last Trade: 82.27 -3.80 -4.42

ALISO VIEJO, Calif. / Dec 23, 2024 / Business Wire / Glaukos Corporation (NYSE: GKOS), an ophthalmic pharmaceutical and medical technology company focused on novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases, today announced the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Epioxa™ (Epi-on), its next-generation corneal cross-linking iLink therapy for the...Read more


MIRA Pharmaceuticals Submits IND for Ketamir-2 to FDA, Marking a Significant Milestone in the Company's Pipeline Development

December 19
Last Trade: 0.83 0.03 3.39

MIAMI, FLORIDA / ACCESSWIRE / December 19, 2024 / MIRA Pharmaceuticals, Inc. (NASDAQ:MIRA), a preclinical-stage pharmaceutical company focused on developing therapies for neurological and neuropsychiatric disorders, today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for Ketamir-2, its novel oral ketamine analog for the treatment of neuropathic pain. The IND...Read more


PTC Therapeutics Announces Vatiquinone NDA Submission to FDA for the Treatment of Children and Adults Living with Friedreich Ataxia

December 19
Last Trade: 39.77 -3.05 -7.12

If approved, vatiquinone would be the first and only authorized therapy for children with FA  PTC's fourth approval application submitted to FDA in 2024  WARREN, N.J., Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug...Read more


Tonix Pharmaceuticals Announces FDA Acceptance of the New Drug Application (NDA) for TNX-102 SL for Fibromyalgia

December 17
Last Trade: 19.71 1.91 10.73

FDA is expected to assign a Prescription Drug User Fee Act (PDUFA) target action date and announce whether Priority Review has been granted in the Day 74 Letter TNX-102 SL is a non-opioid, centrally acting analgesic, granted Fast Track designation by FDA Fibromyalgia affects more than 10 million adults in the U.S. who are mostly women TNX-102 SL has the potential to be the first member of a new class of analgesic drugs for...Read more


Arcutis Biotherapeutics Submits ZORYVE® (roflumilast) Cream 0.05% Supplemental New Drug Application to the FDA for the Treatment of Children Aged 2 to 5 with Mild to Moderate Atopic Dermatitis

December 16
Last Trade: 13.30 -1.00 -6.99

ZORYVE cream 0.05% provided meaningful disease clearance and rapid reduction in itch in pivotal trials Roflumilast cream was well tolerated and demonstrated a favorable safety and tolerability profile for up to 56 weeks of treatment Approximately 1.8 million children with atopic dermatitis (AD) aged 2 to 5 are topically treated in the United States WESTLAKE VILLAGE, Calif., Dec. 16, 2024 (GLOBE NEWSWIRE) -- Arcutis Biotherapeutics,...Read more


Astria Therapeutics Announces FDA Clearance of IND Application for STAR-0310, a Monoclonal Antibody OX40 Antagonist for the Treatment of Atopic Dermatitis

December 10
Last Trade: 3.75 -0.75 -16.67

Phase 1a Trial of STAR-0310 in Healthy Volunteers Expected to Initiate in Q1 2025  Early Proof-of-Concept Results Expected in Q3 2025  BOSTON / Dec 10, 2024 / Business Wire / Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunologic diseases, today announced the U.S. Food and Drug Administration (FDA) clearance of its Investigational New...Read more


NextCure Announces Acceptance of IND Application for LNCB74

December 10
Last Trade: 0.33 -0.06 -14.46

BELTSVILLE, Md., Dec. 10, 2024 (GLOBE NEWSWIRE) -- NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class, and best-in-class therapies to treat cancer, today announced that the U.S. Food and Drug Administration (FDA) accepted an Investigational New Drug (IND) application for initiation of a Phase 1 clinical trial to evaluate LNCB74, a B7-H4-targeting...Read more


Cumberland Pharmaceuticals: FDA Approves Acetadote® sNDA

December 9
Last Trade: 3.91 -0.20 -4.87

New Dosing Regimen Simplifies Administration NASHVILLE, Tenn., Dec. 9, 2024 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on delivering high-quality products to improve patient care, announced today the FDA has approved a supplemental New Drug Application (sNDA) for its Acetadote® (N-acetylcysteine for injection) product. Acetadote is an intravenous (IV) formulation of...Read more


Chimerix to Submit Dordaviprone for Accelerated Approval to U.S. FDA for Patients with Recurrent H3 K27M-Mutant Diffuse Glioma Before Year-End

December 9
Last Trade: 8.51 -0.01 -0.12

Potential Approval in Q3 2025 in Recurrent H3 K27M-Mutant Diffuse Glioma Submission Plan Follows Productive and Collaborative Pre-NDA Interactions with FDA Company to Host Conference Call on Tuesday, December 10 at 8:30 AM ET DURHAM, N.C., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ: CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients...Read more


Crinetics Pharmaceuticals Announces FDA Acceptance of New Drug Application for Paltusotine for Adult Patients with Acromegaly

December 9
Last Trade: 25.56 -0.82 -3.11

SAN DIEGO, Dec. 09, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for investigational candidate paltusotine for the treatment and long-term maintenance therapy of acromegaly in adults. If approved, paltusotine will be the first and only once-daily, oral, selective somatostatin receptor type 2 nonpeptide agonist...Read more


Olema Oncology Announces FDA Clearance of Investigational New Drug Application for OP-3136, a Potent KAT6 Inhibitor

December 9
Last Trade: 3.06 -0.12 -3.77

Phase 1 clinical trial for OP-3136 to initiate in early 2025 SAN FRANCISCO, Dec. 09, 2024 (GLOBE NEWSWIRE) -- Olema Pharmaceuticals, Inc. (“Olema” or “Olema Oncology”, Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational...Read more


Intra-Cellular Therapies Submits Supplemental New Drug Application (sNDA) to FDA for CAPLYTA® (lumateperone) for the Treatment of Major Depressive Disorder as Adjunctive Therapy

December 3
Last Trade: 131.87 0.00 0.00

The sNDA submission is based on positive results from Studies 501 and 502 demonstrating CAPLYTA’s robust antidepressant efficacy and favorable safety and tolerability profile CAPLYTA, if approved as an adjunctive therapy in MDD, would be indicated for the treatment of three different major psychiatry indications affecting over 30 million adult patients in the US BEDMINSTER, N.J., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Intra-Cellular...Read more


Delcath Systems Announces FDA Clearance of IND Application for Phase 2 Clinical Trial of HEPZATO™ in Liver-Dominant Metastatic Colorectal Cancer

December 2
Last Trade: 10.39 -0.52 -4.77

QUEENSBURY, N.Y. / Dec 02, 2024 / Business Wire / Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, today announced that the U.S. Food and Drug Administration (FDA) has completed its 30-day review of the Company's Investigational New Drug (IND) application for a Phase 2 clinical trial evaluating HEPZATO™ in combination with standard of care (SOC) for...Read more


Cytokinetics Announces FDA Acceptance of New Drug Application for Aficamten for the Treatment of Obstructive Hypertrophic Cardiomyopathy

December 2
Last Trade: 34.57 -1.42 -3.95

PDUFA Target Action Date Set for September 26, 2025 SOUTH SAN FRANCISCO, Calif., Dec. 02, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the U.S. Food & Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for aficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (HCM). The FDA assigned the...Read more


Revelation Biosciences Announces FDA Acceptance of Gemini IND

December 2
Last Trade: 3.60 0.89 32.91

Phase 1b Study in CKD Patients to begin early 2025 SAN DIEGO / Dec 02, 2024 / Business Wire / Revelation Biosciences, Inc. (NASDAQ: REVB) (the “Company” or “Revelation”), a clinical-stage life sciences company that is focused on harnessing the power of trained immunity for the treatment of disease, announced today that the United States Food and Drug Administration (FDA) has accepted its investigational new drug (IND) application for...Read more


Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for Vutrisiran for the Treatment of ATTR Amyloidosis with Cardiomyopathy

November 25
Last Trade: 230.19 -2.76 -1.18

Prescription Drug User Fee Act (PDUFA) Date Set for March 23, 2025  CAMBRIDGE, Mass. / Nov 25, 2024 / Business Wire / Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of...Read more


Sangamo Therapeutics Announces U.S. FDA Clearance of IND Application for ST-503 for the Treatment of Idiopathic Small Fiber Neuropathy, a Type of Chronic Neuropathic Pain

November 19
Last Trade: 0.63 -0.08 -11.16

Sangamo plans to initiate enrollment of patients in the Phase 1/2 study for ST-503 in mid-2025 RICHMOND, Calif. / Nov 19, 2024 / Business Wire / Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for its ST-503 program, an investigational epigenetic regulator for the treatment of intractable...Read more


Contineum Therapeutics Expands Clinical Development of PIPE-791 With FDA Authorization of Its Investigational New Drug (IND) Application for Chronic Pain

November 18
Last Trade: 4.79 -0.58 -10.80

Phase 1b study for the potential treatment of osteoarthritis and low back pain expected to commence in the first quarter of 2025 Initial data readout planned for early 2026 Neuropathic component of chronic pain linked to LPA1 activation SAN DIEGO / Nov 18, 2024 / Business Wire / Contineum Therapeutics, Inc. (NASDAQ: CTNM) (Contineum or the Company), a clinical stage biopharmaceutical company focused on discovering and developing...Read more


Arrowhead Pharmaceuticals Submits New Drug Application to U.S. FDA for Plozasiran for the Treatment of Familial Chylomicronemia Syndrome

November 18
Last Trade: 9.99 -0.26 -2.54

The New Drug Application is based on positive results from the Phase 3 PALISADE study People living with familial chylomicronemia syndrome have extremely high triglyceride levels and a substantially higher risk of developing acute pancreatitis and associated long-term complications, including poor quality of life There are currently no FDA approved therapies to treat familial chylomicronemia syndrome PASADENA, Calif. / Nov 18, 2024 /...Read more


Aldeyra Therapeutics Announces FDA Acceptance for Review of Reproxalap New Drug Application for the Treatment of Dry Eye Disease, Expands AbbVie Option Agreement

November 18
Last Trade: 1.92 -0.03 -1.54

PDUFA Date is April 2, 2025 LEXINGTON, Mass. / Nov 18, 2024 / Business Wire / Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmitted New Drug Application (NDA) for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA assigned a...Read more


Elite Pharmaceuticals Receives FDA Approval for Generic Vyvanse

November 18
Last Trade: 0.39 0.04 10.00

Northvale, New Jersey--(Newsfile Corp. - November 18, 2024) - Elite Pharmaceuticals, Inc. (OTCQB: ELTP) ("Elite" or the "Company"), a specialty pharmaceutical company engaged in the development, manufacture, and distribution of niche generic products, today announced that it received approval from the US Food and Drug Administration (FDA) for an Abbreviated New Drug Application (ANDA) for a generic version of Vyvanse® (Lisdexamfetamine...Read more


Vaxcyte Provides Positive Regulatory Updates on VAX-31 Pediatric and Adult Programs

November 12
Last Trade: 30.21 -2.30 -7.07

VAX-31 Infant Indication: Investigational New Drug Application Cleared by FDA; Company Expects to Initiate VAX-31 Infant Phase 2 Study by the End of January 2025 VAX-31 Adult Indication: Breakthrough Therapy Designation Granted by FDA; Company Plans to Initiate Adult Phase 3 Pivotal, Non-Inferiority Study by Mid-2025  VAX-31, Designed to Cover Currently Circulating and Historically Prevalent Strains, is Being Studied for the...Read more


Unicycive Therapeutics Announces U.S. FDA Acceptance of the New Drug Application (NDA) for Oxylanthanum Carbonate (OLC) for the Treatment of Hyperphosphatemia in Patients with Chronic Kidney Disease on Dialysis

November 11
Last Trade: 0.47 0.0092 2.00

FDA sets PDUFA Action Date of June 28, 2025 Company Preparing for 2025 Commercial Launch  LOS ALTOS, Calif., Nov. 11, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company” or “Unicycive”), today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for...Read more


Ionis Pharmaceuticals Announces FDA Acceptance of New Drug Application for Donidalorsen for Prophylactic Treatment of HAE

November 4
Last Trade: 25.51 -2.06 -7.47

Donidalorsen will be a first-in-class RNA-targeted medicine for hereditary angioedema, assuming approval Donidalorsen PDUFA date set for August 21, 2025 Donidalorsen has the potential to be Ionis' second independent commercial launch CARLSBAD, Calif., Nov. 4, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug...Read more


Verastem Oncology Completes Rolling NDA Submission to the FDA for Avutometinib Plus Defactinib as a Treatment for Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer

October 31
Last Trade: 4.65 -0.46 -9.02

Recurrent low-grade serous ovarian cancer is a rare cancer with no FDA-approved treatments Company seeking accelerated approval and priority review of its NDA submission in patients with KRAS mutant low-grade serous ovarian cancer; FDA filing decision expected before the end of 2024 with potential for FDA approval decision by mid-2025 BOSTON / Oct 31, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company...Read more


PTC Therapeutics Announces FDA Acceptance of Translarna™ NDA Resubmission

October 30
Last Trade: 39.77 -3.05 -7.12

WARREN, N.J., Oct. 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the New Drug Application (NDA) for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). "The NDA acceptance for review is a significant milestone that brings us one step closer to providing this important...Read more


Kiora Pharmaceuticals Receives Investigational New Drug Approval to Initiate ABACUS-2, a Phase 2 Clinical Trial of KIO-301 for the Treatment of Retinitis Pigmentosa

October 29
Last Trade: 2.65 0.05 1.92

Encinitas, California--(Newsfile Corp. - October 29, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") today announced it received regulatory approval to initiate a Phase 2 clinical trial to investigate KIO-301 for vision restoration in patients with retinitis pigmentosa. The ABACUS-2 trial will be a 36 patient, multi-center, double-masked, randomized, controlled, multiple dose study enrolling patients with...Read more


Tyra Biosciences Receives IND Clearance from FDA to Proceed with Phase 2 Study of TYRA-300 in Pediatric Achondroplasia (BEACH301)

October 28
Last Trade: 7.06 -0.32 -4.34

TYRA-300 is the first oral FGFR-3 selective inhibitor to be well-tolerated in clinical studies  First child with achondroplasia expected to be dosed in Q1 2025  CARLSBAD, Calif., Oct. 28, 2024 /PRNewswire/ -- Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR)...Read more


IDEAYA Biosciences Announces IND-Clearance for Werner Helicase Development Candidate IDE275 (GSK959) for a Phase 1 Study in MSI-High Solid Tumors

October 28
Last Trade: 14.50 -0.64 -4.23

IDE275 (GSK959) development is progressing into First-in-Human Phase 1 clinical trial(s) for the treatment of MSI-High solid tumors, representing IDEAYA's 5th potential first-in-class clinical program MSI-High prevalence in endometrial, colorectal, and gastric cancers is ~31%, 20%, and 19%, respectively, highlighting the market potential of IDE275 (GSK959) IDEAYA to receive a $7 million payment for IND acceptance, and potential future...Read more


Aligos Therapeutics Announces U.S. FDA Clearance of IND Application for ALG-000184

October 22
Last Trade: 4.71 -0.34 -6.73

Company to conduct a Phase 1 Drug-Drug Interaction Study Phase 2 filing on track for Q1 2025 SOUTH SAN FRANCISCO, Calif., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Aligos Therapeutics, Inc. (Nasdaq: ALGS, “Aligos”), a clinical stage biopharmaceutical company focused on improving patient outcomes through best-in-class therapies for liver and viral diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the...Read more


Amneal Pharmaceuticals Receives U.S. FDA Approval of New Drug Application for Pyridostigmine Bromide Extended-Release Tablets

October 21
Last Trade: 6.99 -0.22 -3.05

Pyridostigmine Bromide Extended Release is a once-daily soman nerve agent pretreatment pyridostigmine product for the U.S. Armed Services and U.S. allies developed utilizing Amneal’s GRANDE® drug delivery technology Developed with the support of Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) BRIDGEWATER, N.J. / Oct 21, 2024 / Business Wire / Amneal Pharmaceuticals, Inc. (Nasdaq:...Read more


LENZ Therapeutics Announces FDA Acceptance of New Drug Application for LNZ100 for the Treatment of Presbyopia

October 21
Last Trade: 18.32 -1.18 -6.05

SAN DIEGO, Oct. 21, 2024 (GLOBE NEWSWIRE) -- LENZ Therapeutics, Inc. (Nasdaq: LENZ or “LENZ” or the “Company”), a pre-commercial biopharmaceutical company focused on the development and commercialization of the first and only aceclidine-based eye drop to improve near vision in people with presbyopia, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for LNZ100 for the...Read more


Tonix Pharmaceuticals Announces Submission of the TNX-102 SL New Drug Application (NDA) for Fibromyalgia to the U.S. Food and Drug Administration (FDA)

October 16
Last Trade: 19.71 1.91 10.73

NDA based on two Phase 3 studies of TNX-102 SL in fibromyalgia with statistically significant results on the primary endpoint of reducing widespread pain; generally well tolerated TNX-102 SL is a non-opioid, centrally acting analgesic, granted Fast Track designation by FDA Fibromyalgia affects more than 10 million adults in the U.S. who are mostly women If approved by FDA, TNX-102 SL would be the first member of a new class of...Read more


Adicet Bio Announces FDA Clearance of IND Amendment to Evaluate ADI-001 in Idiopathic Inflammatory Myopathy and Stiff Person Syndrome

October 16
Last Trade: 0.60 -0.31 -33.95

ADI-001 clinical development program now addresses six autoimmune diseases Patient enrollment for idiopathic inflammatory myopathy and stiff person syndrome cohort expected to be initiated in the first quarter of 2025 Company plans to report initial clinical data from Phase 1 study in multiple autoimmune diseases in the first half of 2025 REDWOOD CITY, Calif. & BOSTON / Oct 16, 2024 / Business Wire / Adicet Bio, Inc. (Nasdaq:...Read more


Cullinan Therapeutics Receives U.S. FDA Clearance of Investigational New Drug Application for CLN-978 Administered Subcutaneously in Patients with Moderate to Severe Systemic Lupus Erythematosus

October 16
Last Trade: 7.32 0.07 0.97

CLN-978 is the first development stage CD19 T cell engager to receive U.S. FDA IND clearance in autoimmune diseases CAMBRIDGE, Mass., Oct. 16, 2024 (GLOBE NEWSWIRE) -- Cullinan Therapeutics, Inc. (Nasdaq: CGEM), a biopharmaceutical company focused on developing modality-agnostic targeted therapies, today announced that the U.S. Food and Drug Administration (FDA) cleared the Company’s Investigational New Drug (IND) Application...Read more


UroGen Pharma Announces FDA Acceptance of its New Drug Application for UGN-102

October 15
Last Trade: 9.66 -0.30 -3.01

PDUFA goal date set for June 13, 2025 UGN-102 would be the first FDA-approved medicine for LG-IR-NMIBC, if approved PRINCETON, N.J. / Oct 15, 2024 / Business Wire / UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced U.S. Food and Drug Administration (FDA) acceptance of the New Drug Application (NDA) for...Read more


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