Ketamine faces a current US drug shortage not expected to abate in the near future1 Current ketamine market estimated at $750 million and projected to reach $3.35 billion globally in 2034.2 NRx anticipates marketing ketamine for all approved uses Company anticipates priority review based on current and anticipated drug shortage NRX-100 to provide innovative, preservative-free IV ketamine formulation to eliminate...Read more
DUBLIN, June 05, 2025 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced today that LUMRYZTM has been granted Orphan Drug Designation (ODD) from the U.S. Food & Drug Administration (FDA) for the treatment of Idiopathic Hypersomnia (IH). Specifically, ODD was granted based on the plausible hypothesis that LUMRYZ may be...Read more
CAMBRIDGE, Mass. / Jun 04, 2025 / Business Wire / Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. Food & Drug Administration. “This...Read more
Aortic Stenosis is a Serious and Widespread Condition; The AI-ECG Algorithm Offers a Powerful Diagnostic Solution Designed for Seamless Integration with Hospital EHR Systems Southlake, TX, June 04, 2025 (GLOBE NEWSWIRE) -- HeartSciences Inc. (Nasdaq: HSCS; HSCSW) (“HeartSciences” or the “Company”), an artificial intelligence (“AI”) powered medical technology company focused on advancing the capabilities of ECGs/EKGs for the earlier...Read more
Designation Supported by CLOVER WaM Phase 2 Study Data Which Reported an 83.6% Overall Response Rate (ORR) Seeking Guidance from EMA to Determine if CLOVER WaM Phase 2 Data Meets Criteria to Apply for Fast-Track, Conditional Marketing Authorization, Answer Expected Late July FLORHAM PARK, N.J., June 04, 2025 (GLOBE NEWSWIRE) -- Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on...Read more
Nubeqa is the first and only in the U.S. and FDA-approved androgen receptor inhibitor (ARi) for the treatment of patients with hormone-sensitive prostate cancer (mHSPC), in combination with androgen deprivation therapy (ADT), with or without chemotherapy This third approval is based on positive results from the pivotal Phase III ARANOTE trial and broadens the indication profile of darolutamide in mHSPC, enabling its use in combination...Read more
PALO ALTO, Calif. / Jun 03, 2025 / Business Wire / Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, today announced that its Shield multi-cancer detection (MCD) test has been granted Breakthrough Device designation from the U.S. Food and Drug Administration (FDA). The Shield MCD test is a methylation-based blood test for the multi-cancer screening of multiple cancer types including bladder, colorectal, esophageal,...Read more
AMX0114 is an Amylyx-developed antisense oligonucleotide designed to target calpain-2, a key contributor to the axonal degeneration pathway in ALS Phase 1 LUMINA trial of AMX0114 underway; early cohort data expected in 2025 CAMBRIDGE, Mass. / Jun 03, 2025 / Business Wire / Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track...Read more
CAMBRIDGE, Mass., June 03, 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 granted orphan drug designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). “Sickle cell disease is a...Read more
Authorized expanded access for ANKTIVA to treat lymphopenia, a life-threatening immune deficiency induced by chemotherapy, radiotherapy, and immunotherapy with depletion of natural killer (NK) and CD4+ CD8+ T cells (lymphocytes) Expanded Access includes all patients with solid tumors who have failed first-line treatment on chemotherapy, radiotherapy or immunotherapy and exhibit low Absolute Lymphocyte Counts (ALC...Read more
This major Swoop® system scanner redesign and Optive AI™ software elevate the AI-powered portable MRI experience for clinicians and their patients across multiple sites of care. GUILFORD, Conn. / Jun 02, 2025 / Business Wire / Hyperfine, Inc. (Nasdaq: HYPR), the groundbreaking health technology company that has redefined brain imaging with the first FDA-cleared AI-powered portable MRI system for the brain—the Swoop® system—announced...Read more
Once-weekly TransCon CNP demonstrated significantly higher annualized growth velocity, the primary endpoint, compared to placebo Multiple benefits beyond linear growth were observed compared to placebo, with a safety and tolerability profile similar to placebo The FDA grants Priority Review designation for the evaluation of medicines that, if approved, would provide a significant improvement in the safety or effectiveness of the...Read more
Both ERAS-4001 and potential best-in-class pan-RAS molecular glue ERAS-0015 received IND clearance in May ahead of company guidance Initial Phase 1 monotherapy data for both RAS-targeting programs expected in 2026 SAN DIEGO, June 02, 2025 (GLOBE NEWSWIRE) -- Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK...Read more
New Drug Application based on positive results from the Phase 2 KOMET-001 trial FDA assigns a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 Potential first approval of a menin inhibitor for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation SAN DIEGO and TOKYO, June 01, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd....Read more
mNEXSPIKE becomes Moderna's third FDA-approved product CAMBRIDGE, MA / ACCESS Newswire / May 31, 2025 / Moderna, Inc. (NASDAQ:MRNA) today announced that the U.S. Food and Drug Administration (FDA) has approved mNEXSPIKE® (mRNA-1283), a new vaccine against COVID-19, for use in all adults 65 and older, as well as individuals aged 12-64 years with at least one or more underlying risk factor as defined by the Centers for Disease Control...Read more
CAMBRIDGE, Mass., May 29, 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 granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the...Read more
WINTER SPRINGS, Fla., May 29, 2025 (GLOBE NEWSWIRE) -- Iradimed Corporation (NASDAQ: IRMD), a global leader in innovative medical devices for MRI environments, today announced that the U.S. Food and Drug Administration (FDA) has granted 510(k) clearance for its next-generation MRidium® 3870 IV Infusion Pump System. This advanced, MRI-compatible infusion pump extends Iradimed’s unique position as the world’s only supplier of non-magnetic...Read more
KHINDIVI is the first and only FDA-approved hydrocortisone oral solution Commercial launch expected the week of June 2nd Eton expects combined peak sales of KHINDIVI and ALKINDI SPRINKLE® (hydrocortisone) oral granules to exceed $50 million DEER PARK, Ill., May 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and...Read more
In pivotal Phase 3 trials, TRYPTYR demonstrated rapid natural tear production as early as Day 11-2 TRYPTYR is a first-in-class TRPM8 receptor agonist that rapidly stimulates natural tear production in patients with Dry Eye Disease (DED)3 Approximately 38 million individuals in the U.S. are living with DED, yet less than 10% of diagnosed patients are being treated with a prescription product4 GENEVA / May 28, 2025 / Business Wire /...Read more
Latest Swoop® System software delivers a substantial leap in image quality for AI-powered portable MR brain imaging. GUILFORD, Conn. / May 28, 2025 / Business Wire / Hyperfine, Inc. (Nasdaq: HYPR), the groundbreaking health technology company that has redefined brain imaging with the world’s first FDA-cleared AI-powered portable MRI system for the brain—the Swoop® system—today announced FDA clearance of its next-generation software....Read more
NEEDHAM, Mass., May 28, 2025 (GLOBE NEWSWIRE) -- Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical-stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to CAN-2409 (aglatimagene besadenovec), the Company’s...Read more
Fast Track designation granted for investigational TEV-53408, an anti-IL-15 antibody Designation underscores the potential of TEV-53408, currently undergoing a Phase 2a study for the treatment of celiac disease Candidate demonstrates Teva’s commitment to bringing important new treatment options to underserved patient populations TEL AVIV, Israel, May 27, 2025 (GLOBE NEWSWIRE) -- Teva Pharmaceutical Industries, Ltd. (NYSE and TASE:...Read more
The Tendyne system replaces mitral valves that are not functioning properly due to a buildup of calcium in the base of the valves, known as severe mitral annular calcification (MAC) Tendyne offers a new treatment option for patients who are not candidates for open-heart surgery or mitral repair This approval expands Abbott's portfolio of minimally invasive structural heart therapies in the U.S. ABBOTT PARK, Ill., May 27, 2025...Read more
LANGHORNE, Pa. / May 27, 2025 / Business Wire / Savara Inc. (Nasdaq: SVRA) (the Company), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, today announced that the Company received an RTF letter from the FDA for the BLA of MOLBREEVI as a therapy to treat patients with autoimmune PAP. Upon preliminary review, the FDA determined that the BLA submitted in March 2025 was not sufficiently complete to permit...Read more
NEWTOWN, Pa., May 27, 2025 (GLOBE NEWSWIRE) -- Traws Pharma, Inc. (NASDAQ: TRAW) (“Traws Pharma”, “Traws” or “the Company”), a clinical-stage biopharmaceutical company developing novel therapies to target critical threats to human health from respiratory viral diseases, today announced receipt of written responses to questions submitted for a Type B pre-Investigational New Drug Application meeting with the U.S. Food and Drug...Read more
HADLIMA™ (adalimumab-bwwd) injection, 40 mg/0.4 mL & 40 mg/0.8 mL is now interchangeable with all high- and low-concentration presentations (autoinjector, prefilled syringe, and single-dose vial) of Humira (adalimumab)1,2 The interchangeability designation for HADLIMA is based on a Pharmacokinetics, Efficacy, Safety, and Immunogenicity study of SB5 versus Humira in patients with moderate to severe chronic plaque psoriasis3 An...Read more
MALVERN, Pa., May 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the United States Food and Drug Administration (U.S. FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod...Read more
CAMBRIDGE, MA / ACCESS Newswire / May 23, 2025 / Moderna, Inc. (NASDAQ:MRNA) today announced that it has submitted an application to the U.S. Food and Drug Administration (FDA) for review of its Spikevax 2025-2026 formula, targeting the SARS-CoV-2 variant LP.8.1. The submission is based on guidance from the U.S. FDA, which advised that COVID-19 vaccines should be updated to a monovalent JN.1 lineage, with a preference for the LP.8.1...Read more
FDA’s approval of YUTREPIA paves the way for prescribers to add a new treatment option for patients with PAH and PH-ILD YUTREPIA is designed to enhance deep-lung delivery with an easy-to-use device requiring low inspiratory effort Demonstrated tolerability and titratability in the pivotal INSPIRE study Liquidia will host a webcast Tuesday, May 27, 2025 at 8:30 a.m. ET to provide an update on commercial launch...Read more
Nucala is the only approved biologic studied in a wide COPD population with an eosinophilic phenotype characterized by blood eosinophil count (BEC) starting at 150 cells/μL Approval based on the positive MATINEE and METREX phase III trials MATINEE data included reduction of exacerbations leading to hospitalization and/or emergency department visits Nearly 70% of patients in the US who are inadequately controlled on inhaled triple...Read more
Once-daily ZORYVE foam provides powerful clearance and rapid itch relief from head to toe with no limitation on duration of use More than half of the nearly 9 million people in the United States with plaque psoriasis experience scalp involvement Healthcare providers and individuals with plaque psoriasis can now choose ZORYVE foam or cream to treat plaque psoriasis anywhere it appears on the body Fifth FDA approval for ZORYVE in less...Read more
Susvimo can help people with diabetic retinopathy (DR) maintain their vision and prevent progression to blindness with only one treatment every nine months Susvimo’s innovative technology via the Port Delivery Platform may offer an alternative to regular eye injections in the US Diabetic retinopathy affects almost 10 million people in the US and is the third FDA-approved indication for Susvimo, which is also approved for treating wet,...Read more
Study CL-QRX003-002 will test whole-body application of QRX003 in conjunction with off-label systemic therapy in approximately 12-15 subjects. Study CL-QRX003-002 complements first whole-body pivotal study, CL-QRX003-003, being led by Dr. Amy Paller at Northwestern University and supported by up to six international sites that are expected to open, where subjects are being tested with QRX003 as monotherapy. Combined data from Studies...Read more
GENEVA, SWITZERLAND / ACCESS Newswire / May 22, 2025 / RELIEF THERAPEUTICS Holding SA (SIX:RLF) (OTCQB:RLFTF) (OTCQB:RLFTY ) (Relief or the Company), a biopharmaceutical company committed to delivering innovative treatment options for select specialty, unmet and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to RLF-TD011 for the treatment of...Read more
PRINCETON, N.J. / May 21, 2025 / Business Wire / UroGen Pharma Ltd. (Nasdaq: URGN), a leading biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, announced the outcome of today’s meeting of the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA), which discussed the new drug application (NDA) for investigational drug UGN-102...Read more
Designation is based on the pivotal Phase 3 DRAGON trial interim analysis results demonstrating Tinlarebant’s efficacy and favorable safety profile Trial completion expected by Q4 2025 (including a three-month follow-up period) Tinlarebant has previously been granted Fast Track and Rare Pediatric Disease Designations in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and the Pioneer Drug Designation in Japan for...Read more
ODAC recommendation based on the positive progression-free survival and clinical benefit in the Phase 3 AQUILA study If approved, DARZALEX FASPRO® would be the first treatment to potentially delay or prevent progression to multiple myeloma RARITAN, N.J., May 20, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) announced today the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) voted (6-2) in...Read more
Columvi is the first bispecific antibody to show a statistically significant and clinically meaningful 41% survival benefit in R/R DLBCL in the Phase III STARGLO study There is an urgent need for effective, immediately available therapies that are broadly accessible to people with transplant-ineligible R/R DLBCL This first-of-its-kind Columvi combination could provide a much-needed, off-the-shelf and fixed-duration treatment option for...Read more
Stryker's first basivertebral nerve ablation system targets the basivertebral nerve to provide relief from chronic* vertebrogenic low back pain. PORTAGE, Mich., May 19, 2025 /PRNewswire/ -- Stryker (NYSE:SYK), a global leader in medical technologies, announced that its OptaBlate basivertebral nerve ablation system (OptaBlate BVN) received 510(k) clearance from the U.S. Food and Drug Administration. OptaBlate BVNA is used in a...Read more
TAMPA, FL, May 19, 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 that the U.S. Food and Drug Administration (FDA) has listed U.S....Read more
Investor call and webcast scheduled for today at 8:30 a.m. ET NEW YORK, May 19, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company to initiate its Phase 3b clinical trial of NurOwn® (autologous MSC-NTF cells) for the treatment of amyotrophic...Read more
Nuvaxovid™ is the only recombinant protein-based, non-mRNA COVID-19 vaccine available in the U.S. Biologics License Application approval triggers $175 million Sanofi milestone payment, with additional COVID-19 vaccine milestones and ongoing tiered royalties to be recognized when earned GAITHERSBURG, Md., May 19, 2025 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX) today announced that the U.S. Food and Drug Administration...Read more
The Company is on track to initiate a pivotal study for ZL-1310 in small cell lung cancer (SCLC) in 2025 SHANGHAI & CAMBRIDGE, Mass. / May 19, 2025 / Business Wire / Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ZL-1310, the Company’s potential first-in-class Delta-like ligand (DLL3) antibody-drug conjugate (ADC), for the treatment of...Read more
Regulatory alignment with FDA confirmed, clearing path to pivotal U.S. clinical trial 150-page strategic business plan and advanced financial models completed Investor outreach launched to fund clinical execution and market entry Vancouver, British Columbia and Sacramento, California--(Newsfile Corp. - May 16, 2025) - Izotropic Corporation (CSE: IZO) (OTCQB: IZOZF) (FSE: 1R3) ("Izotropic" or the "Company"), a medical device company...Read more
TORONTO, May 16, 2025 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (OTCQB: PHRRF) (CSE: PHRM) ("PharmaTher" or the "Company"), a specialty pharmaceutical company focused on ketamine for unmet medical needs, today announced the U.S. Food and Drug Administration (FDA) has extended the approval goal date for the review of its new drug application for ketamine. The new approval goal date is August 9, 2025, revised from the previous date of...Read more
PDUFA target action date of January 13, 2026 If approved, FILSPARI would be the first and only FDA-approved treatment for FSGS, a rare kidney condition and a leading cause of kidney failure SAN DIEGO / May 15, 2025 / Business Wire / Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for traditional approval of FILSPARI...Read more
Zynyz® (retifanlimab-dlwr), a PD-1 inhibitor, is now approved for the treatment of advanced squamous cell carcinoma of the anal canal (SCAC) in combination with chemotherapy and as a monotherapy Patients with SCAC treated with Zynyz achieved a statistically significant improvement in progression-free survival and improvement in overall survival WILMINGTON, Del. / May 15, 2025 / Business Wire / Incyte (Nasdaq:INCY) today announced that...Read more
Brekiya® becomes the first and only DHE autoinjector that allows patients to self-administer the same medication used in hospitals in a ready-to-use form Product will be available for appropriate patients in the second half of 2025 BRIDGEWATER, N.J., May 15, 2025 (GLOBE NEWSWIRE) -- Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has...Read more
NEW YORK, May 14, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has received seven years of orphan-drug exclusive approval from U.S. Food and Drug Administration (FDA) for Ryoncil® (remestemcel-L) for treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older. This...Read more
Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. Troriluzole has been granted Fast-Track, Orphan Drug Designation (ODD) and Priority Review from the FDA. Troriluzole would be the first and only FDA-approved treatment for SCA, if approved. NEW HAVEN, Conn., May 14, 2025 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), today...Read more
EMRELIS is the first and only treatment approved for previously treated advanced NSCLC patients with high c-Met protein overexpression who often face poor prognosis and have limited treatment options Lung cancer remains the leading cause of cancer-related deaths throughout the world1 NORTH CHICAGO, Ill., May 14, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that EMRELIS™ (telisotuzumab vedotin-tllv) has been granted...Read more
WELIREG becomes the only approved and available treatment in the U.S. for eligible patients with advanced PPGL RAHWAY, N.J. / May 14, 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 approved WELIREG® (belzutifan), Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, for the treatment of adult and pediatric...Read more
PARAMUS, N.J., May 14, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S. Food and Drug Administration has accepted for review the Biologics License Application (BLA) submission by REGENXBIO Inc. (REGENXBIO; Headquarters: Rockville, Maryland, USA; CEO: Curran M. Simpson, NASDAQ: RGNX) for RGX-121 (clemidsogene lanparvovec), a potential first-in-class,...Read more
FDA grants Priority Review of application, with PDUFA target action date of September 12, 2025 ORLADEYO would be the first targeted oral prophylactic therapy for patients with HAE under the age of 12, if approved RESEARCH TRIANGLE PARK, N.C., May 14, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application...Read more
The latest approval will help provide clearer echocardiogram images for cardiologists to diagnose heart conditions in pediatric patients Optison is the only polyethylene glycol (PEG)-free ultrasound enhancing agent available in the U.S. ARLINGTON HEIGHTS, Ill. / May 13, 2025 / Business Wire / GE HealthCare (Nasdaq: GEHC) today announced the U.S. Food and Drug Administration (FDA) has approved a pediatric indication for the company’s...Read more
The AYON Body Contouring System is the first FDA cleared all-in-one platform for the aesthetic surgical suite Plan to initiate the commercial launch of the AYON Body Contouring System to key opinion leader surgeons in critical geographies during the second half of 2025 CLEARWATER, Fla., May 13, 2025 (GLOBE NEWSWIRE) -- Apyx Medical Corporation (NASDAQ:APYX) (“Apyx Medical”; the “Company”), the manufacturer of a proprietary helium...Read more
FDA assigns PDUFA target action date of November 9, 2025 RGX-121 on track to be the first gene therapy and one-time treatment for MPS II Partner Nippon Shinyaku to lead commercialization upon potential approval REGENXBIO to lead commercial manufacturing and supply chain ROCKVILLE, Md., May 13, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the U.S. Food and Drug Administration (FDA) has...Read more
Company advancing plans to develop TPI 287, a novel, late stage abeotaxane that appears to cross the blood-brain barrier and has published clinical efficacy data in glioblastoma multiforme (GBM) Management releases "What This Means" segment discussing the receipt of Orphan Drug Designations for TPI 287; Access here HOUSTON, TX / ACCESS Newswire / May 13, 2025 / CNS Pharmaceuticals, Inc. (NASDAQ:CNSP) ("CNS" or the "Company"), a...Read more
CAMBRIDGE, Mass., May 12, 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 granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base...Read more
ENGLEWOOD, Colo., May 12, 2025 /PRNewswire/ -- Zynex, Inc. (NASDAQ: ZYXI), an innovative medical technology company specializing in the manufacture and sale of non-invasive medical devices for pain management, rehabilitation, and patient monitoring, today announced it has submitted a 510(k) application to the U.S. Food and Drug Administration ("FDA") for its NiCO™, Noninvasive CO-Oximeter device. This submission marks a...Read more
Drug candidate LP-184 to be evaluated in combination with immune checkpoint inhibitors in biomarker-defined NSCLC patients with KEAP1 and/or STK11 mutations and low PD-L1 expression. The FDA has cleared the LP-184 investigational new drug (IND) application amendment for the treatment of a biomarker defined patient population of non-small cell lung cancer (NSCLC). The planned phase 1b/2 clinical trial plans to evaluate LP-184 in advanced...Read more
This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD as a one-time treatment for Parkinson’s disease RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review LONDON and NEW YORK, May 09, 2025 (GLOBE...Read more
AVMAPKI plus FAKZYNJA to be commercially available by prescription as a convenient oral combination co-packaged together and will be known as “AVMAPKI FAKZYNJA CO-PACK” Accelerated approval, well ahead of the June 30, 2025 PDUFA action date, was based on the Phase 2 RAMP 201 study that demonstrated a 44% overall response rate in patients with KRAS mutant recurrent LGSOC Verastem to host investor conference call and webcast today at 2:30...Read more
Meeting to Take Place on July 25, 2025 Meeting’s Purpose is To Discuss Upcoming Clinical Development Plan and Protocol Designs GLEN ALLEN, Va., May 08, 2025 (GLOBE NEWSWIRE) -- Adial Pharmaceuticals, Inc. (NASDAQ: ADIL) (“Adial” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment and prevention of addiction and related disorders, today announced that the FDA has granted...Read more
Breakthrough Therapy Designation request based on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response Veligrotug met all of its primary and secondary endpoints in the pivotal THRIVE and THRIVE-2 clinical trials in active and chronic TED First and only drug candidate in chronic TED to demonstrate statistically significant and clinically...Read more
PRINCETON, N.J. / May 07, 2025 / Business Wire / UroGen (NASDAQ: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced that the U.S. Food and Drug Administration (FDA) has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to review the new drug application (NDA) for UGN-102 (mitomycin) for intravesical solution, an...Read more
SEATTLE / May 06, 2025 / Business Wire / Omeros Corporation (Nasdaq: OMER) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the Biologics License Application (BLA) for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). The resubmission was classified as a Class 2 resubmission and pursuant to the Prescription Drug User...Read more
Targeting to evaluate IDE849 (SHR-4849) in SCLC, NETs, and other DLL3-upregulated solid tumors, and in combination with IDE161/PARG to potentially enhance durability Targeting to present clinical data of over 40 SCLC patients, including the dose escalation and expansion, from partner Hengrui at medical conference in Q3 2025 SOUTH SAN FRANCISCO, Calif., May 6, 2025 /PRNewswire/ -- IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision...Read more
FDA lifts clinical hold enabling resumption of clinical trials FDA has granted a Type A Meeting to discuss path forward for BLA THOUSAND OAKS, Calif. / May 05, 2025 / Business Wire / Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today announced...Read more
The FDA has cleared the investigational new drug (IND) application of LP-184 in triple negative breast cancer (TNBC) treatment – a novel small molecule advanced and developed with Lantern’s AI platform, RADR® The phase 1b/2 clinical trial plans to evaluate LP-184 in recurrent TNBC patients as both monotherapy and in combination with olaparib. This achievement follows the FDA’s Orphan Drug Designation for LP-184 in TNBC in 2023 and FDA...Read more
Registrational study in patients with tumor hyperinsulinism (HI) expected to commence mid-year Designation underscores need for therapies to treat severe hypoglycemia in the oncology setting REDWOOD CITY, Calif., May 05, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage rare disease company focused on treating hypoglycemia caused by HI, today announced that the U.S. Food and Drug...Read more
WASHINGTON, May 5, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) informed Vanda that the New Drug Application (NDA) for Bysanti™ (milsaperidone) has been filed, and that at this time no potential review issues have been identified. The FDA has set February 21, 2026 as the target date for decision on this application. Bysanti™ is a new chemical...Read more
Company remains on track for PDUFA target action date of August 31, 2025 Advisory committee meeting to be held in advance of target action date SAN DIEGO, May 05, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of a mid-cycle review meeting with the U.S. Food...Read more
The Company is now recognized by the U.S. FDA to support the cross-border supply of GMP MDMA and psilocybin to authorized entities in the United States. Vancouver, British Columbia--(Newsfile Corp. - May 5, 2025) - Optimi Health Corp. (CSE: OPTI) (OTCQX: OPTHF) (FSE: 8BN) ("Optimi" or the "Company"), a manufacturer of GMP-certified MDMA and psilocybin, has received its U.S. FDA Establishment Identifier (FEI) number—a key regulatory...Read more
Fast Track Designation highlights potential of ATH434 to address high unmet need for individuals with MSA MELBOURNE, Australia and SAN FRANCISCO, May 05, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug...Read more
The U.S. FDA has approved SELARSDI™ (ustekinumab-aekn) as interchangeable with the reference biologic Stelara® (ustekinumab) in all presentations matching the reference product, effective as of April 30, 2025 SELARSDI is approved for all indications matching the reference product SELARSDI is indicated for the treatment of moderate to severe plaque psoriasis and active psoriatic arthritis in adults and pediatric patients 6 years and...Read more
HOUSTON, TX, May 01, 2025 (GLOBE NEWSWIRE) -- Nexalin Technology, Inc. (Nasdaq: NXL; NXLIW) (the “Company” or “Nexalin”), today announced that it plans to submit a Q-Submission (“Q-Sub”) to the U.S. Food and Drug Administration (FDA) related to its Gen-2 SYNC system. This regulatory milestone is intended to facilitate structured dialogue with the FDA regarding clinical trial design for the treatment of Alzheimer’s disease, dementia, and...Read more
SUNNYVALE, Calif., May 01, 2025 (GLOBE NEWSWIRE) -- Intuitive (NASDAQ: ISRG), a global technology leader in minimally invasive care and the pioneer of robotic-assisted surgery, announced today that the U.S. Food and Drug Administration (FDA) cleared the da Vinci Single Port (SP) surgical system for transanal local excision/resection, a form of minimally invasive surgery performed through a natural orifice to avoid abdominal surgical...Read more
Transforming Care for Severe AS Patients IRVINE, Calif. / May 01, 2025 / Business Wire / Edwards Lifesciences (NYSE: EW) announced today that the U.S. Food and Drug Administration (FDA) has approved its transcatheter aortic valve replacement (TAVR) therapy, the SAPIEN 3 platform, for severe aortic stenosis (AS) patients without symptoms, marking the first FDA approval for TAVR in asymptomatic patients. Approval of the SAPIEN 3...Read more
EMERYVILLE, Calif., May 01, 2025 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented benefits to patients, today announced that the U.S. Food and Drug Administration (FDA) has granted the Regenerative Medicine Advanced Therapy (RMAT)...Read more
LONDON and NEW YORK, May 01, 2025 (GLOBE NEWSWIRE) -- OKYO Pharma Limited (NASDAQ: OKYO), an ophthalmology-focused bio-pharmaceutical company which is developing urcosimod (formerly called OK-101) to treat NCP, an ocular condition associated with chronic and often severe pain but without an FDA-approved therapy, announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to urcosimod for the treatment of...Read more
Decision to submit based on a positive overall response rate (ORR) (p-value < 0.0001), one of the dual primary endpoints in the Phase 3 EPCORE® FL-1 trial Full results from the trial will be submitted for presentation at an upcoming medical conference in 2025 COPENHAGEN, Denmark / May 01, 2025 / Business Wire / Genmab A/S (Nasdaq: GMAB) announced today its intention to submit in the first half of 2025 a supplemental Biologics...Read more
First and only FcRn blocker approved in anti-AChR and anti-MuSK antibody positive adults and pediatric gMG patients aged 12 and older IMAAVY delivered rapid and substantial reduction in immunoglobulin G (IgG) levels, one of the root causes of gMG, in both the adult and pediatric pivotal studies gMG patients taking IMAAVY demonstrated 20 months of lasting disease...Read more
XORTX will focus on key steps to advance a NDA filing for Gout indication CALGARY, Alberta, April 30, 2025 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late stage clinical pharmaceutical company focused on developing innovative therapies to treat gout and progressive kidney disease, is pleased to announce it has received responses from the US Food...Read more
WALTHAM, Mass., April 30, 2025 /PRNewswire/ -- Q32 Bio Inc. (Nasdaq: QTTB) ("Q32 Bio"), a clinical stage biotechnology company focused on developing innovative therapies for alopecia areata (AA) and other autoimmune and inflammatory diseases, today announced that the United States Food and Drug Administration ("FDA") has granted Fast Track designation (FTD) to Q32 Bio's bempikibart (ADX-914) for the treatment of AA. Bempikibart is a fully...Read more
WAYNE, Pa., April 30, 2025 (GLOBE NEWSWIRE) -- Teleflex Incorporated (NYSE: TFX), a leading global provider of medical technologies, today announced U.S. Food and Drug Administration (FDA) 510(k) clearance of an expansion to the Indications for Use of the QuikClot Control+™ Hemostatic Device to include all grades of internal and external bleeding. The QuikClot Control+™ Device was previously indicated for temporary control of class III...Read more
RINVOQ (upadacitinib) is now the first and only oral Janus Kinase (JAK) inhibitor approved for the treatment of GCA in adults Also known as temporal arteritis, GCA is the most common vasculitis affecting adults in Western countries2 This marks the ninth approved indication for RINVOQ in the U.S., across rheumatology, gastroenterology, and dermatology1 NORTH CHICAGO, Ill., April 29, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) today...Read more
ZEVASKYN fills a critical need for people living with RDEB, a debilitating dermatological condition with no cure Approval based on the pivotal Phase 3 VIITAL™ study, showing ZEVASKYN resulted in significant wound healing and pain reduction after a single treatment with a favorable safety profile ZEVASKYN to be available through Qualified Treatment Centers beginning in 3Q 2025 Abeona Assist™ patient services program...Read more
QUEENSBURY, N.Y. / Apr 28, 2025 / 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
NDA submitted for the treatment of central diabetes insipidus Expected 10-month review; commercial preparations underway for a potential Q1 2026 launch DEER PARK, Ill., April 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the submission of a New Drug...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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