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
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
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
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
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
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
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
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
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
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
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 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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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 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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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 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
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
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
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
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
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
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
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
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
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
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-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
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
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
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
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
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
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
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
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
sNDA is Based on Full Findings from the Positive HELIOS-B Phase 3 Study Priority Review Voucher Utilized to Accelerate Review Period Alnylam to Host and Webcast TTR Investor Day Event Today in New York City CAMBRIDGE, Mass. / Oct 09, 2024 / Business Wire / Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the submission of its supplemental New Drug Application...Read more
KT-621 has demonstrated dupilumab-like activity and was well tolerated in a wide variety of preclinical models of TH2 diseases KT-621 is expected to start Phase 1 in October, with Phase 1 data in the first half of 2025 WATERTOWN, Mass., Oct. 09, 2024 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of small molecule medicines using targeted protein...Read more
Approval allows for larger field treatment of actinic keratosis (AK) on face and scalp with Ameluz®-PDT using the BF-RhodoLED or the RhodoLED XL lamp Supplemental New Drug Application (sNDA) supported by two Phase 1 safety studies AK is the second most common diagnosis made by dermatologists in the United States1 An estimated 13 million treatments given each year for AK in the US2 WOBURN, Mass., Oct. 07, 2024 (GLOBE NEWSWIRE) --...Read more
Based on ECHO Phase III trial which demonstrated CALQUENCE combination reduced risk of disease progression or death by 27% compared to standard-of-care chemoimmunotherapy Submission to be reviewed under Project Orbis WILMINGTON, Del. / Oct 03, 2024 / Business Wire / AstraZeneca’s supplemental New Drug Application (sNDA) for CALQUENCE® (acalabrutinib) has been accepted and granted Priority Review in the US for the treatment of adult...Read more
LEXINGTON, Mass. / Oct 03, 2024 / Business Wire / Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated and metabolic diseases, today announced the resubmission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for topical ocular reproxalap, an investigational new drug candidate, for the treatment...Read more
First program to combine Recursion’s end-to-end suite of AI-enabled active learning modules, resulting in target identification to IND enabling studies in under 18 months Plan to initiate dosing of Phase 1/2 in Q4 2024 to evaluate REC-1245 in a biomarker enriched patient population, including patients with solid tumors and lymphoma SALT LAKE CITY, Oct. 02, 2024 (GLOBE NEWSWIRE) -- Recursion (NASDAQ: RXRX), a leading clinical stage...Read more
WARREN, N.J., Oct. 1, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU). A Prescription Drug User Fee Act (PDUFA) target action date is expected to be provided in the Day 74 Letter. "The FDA filing acceptance for sepiapterin is a critical milestone...Read more
ClearPoint Neuro is a global therapy-enabling platform company providing stereotactic navigation and delivery to the brain. Applications of our ClearPoint Neuro Navigation System include electrode lead placement, placement of catheters, and biopsy. The platform has FDA clearance and is...
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