FDA
Jul 22, 2025
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths; ImCheck’s ICT01 Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia; RedHill Biopharma Gets Positive FDA Feedback on RHB-204 for Crohn’s Disease Approval Pathway; FDA Grants Fast Track Designation to ProMIS Neurosciences’ PMN310 for Alzheimer’s Disease; Oruka Therapeutics Receives IND Clearance for Phase IIa Trial of ORKA-001 in Psoriasis
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths Sarepta Therapeutics, Inc. has voluntarily and temporarily paused all shipments of ELEVIDYS (delandistrogene moxeparvovec) in the U.S., effective July 22, 2025, following engagement with the FDA on safety labeling u...
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Jul 15, 2025
Nanoscope’s MCO-010 Begins FDA Rolling Submission for Retinitis Pigmentosa; Bayer’s KERENDIA Approved by FDA for Heart Failure With Preserved Ejection Fraction; Klotho Neurosciences’ KLTO-202 Receives FDA Orphan Drug Designation for ALS; Trethera’s TRE-515 Granted FDA Fast Track Designation for Metastatic Prostate Cancer; Adcentrx’s ADRX-0405 Awarded FDA Orphan Drug Designation for Gastric Cancer
Nanoscope Therapeutics Begins FDA Rolling Submission for MCO-010 in Retinitis Pigmentosa Nanoscope Therapeutics has initiated a rolling submission of its Biologics License Application (BLA) to the FDA for MCO-010, a first-in-class, gene-agnostic optogenetic therapy targeting severe vision loss due to retinitis p...
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Jul 08, 2025
Merck’s WINREVAIR Granted FDA Priority Review for Pulmonary Arterial Hypertension; KalVista’s EKTERLY Approved by FDA as First Oral On-Demand Therapy for Hereditary Angioedema; Fujifilm’s FF-10832 Receives FDA Orphan Drug Designation for Biliary Tract Cancer; Mustang Bio’s MB-101 Granted FDA Orphan Drug Designation for Glioblastoma and Astrocytomas; Denali’s Tividenofusp Alfa Accepted for FDA Priority Review for Hunter Syndrome
Merck's WINREVAIR Gets FDA Priority Review for Pulmonary Arterial Hypertension Merck, also known as MSD outside the U.S. and Canada, announced that the FDA has accepted and granted Priority Review to the supplemental Biologics License Application (sBLA) for WINREVAIR (sotatercept-csrk). The application seeks to ...
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Jul 02, 2025
How the Amniotic Membrane Market is Transforming Regenerative Healthcare Worldwide
The amniotic membrane market is revolutionizing healthcare by harnessing the natural healing power of the amniotic membrane, a thin placental layer renowned for its regenerative properties. This remarkable biomaterial is transforming treatments across specialties, particularly in amniotic membrane eye treatment, wh...
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Jul 01, 2025
Sobi’s GAMIFANT Approved by FDA as First Treatment for Macrophage Activation Syndrome in Still’s Disease; GSK Cuts RTS,S Malaria Vaccine Price for Children in Endemic Regions to Under $5; Schrödinger’s SGR-1505 Receives FDA Fast Track Designation for Relapsed/Refractory Waldenström Macroglobulinemia; Ultragenyx’s GTX-102 Granted FDA Breakthrough Therapy Designation for Angelman Syndrome; Eli Lilly’s AMYVID Label Updated by FDA to Support Alzheimer’s Disease Diagnosis
FDA Approves Sobi’s GAMIFANT as the First Treatment for Macrophage Activation Syndrome in Still’s Disease In a major advancement for rare inflammatory conditions, the FDA has approved GAMIFANT (emapalumab-lzsg) for the treatment of hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MA...
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Jun 24, 2025
Incyte’s MONJUVI Combo Approved by FDA for Relapsed/Refractory Follicular Lymphoma; Gilead’s YEZTUGO Becomes First FDA-Approved HIV Prevention with 6-Month Protection; FDA Reviewing Incyte’s OPZELURA for Pediatric Atopic Dermatitis (Ages 2–11); Archeus Technologies Receives FDA Clearance for Prostate Cancer Therapy ART-101; Cycle Pharmaceuticals’ HARLIKU Gets First FDA Approval for Alkaptonuria
Incyte’s MONJUVI Combo Approved by FDA for Relapsed/Refractory Follicular Lymphoma Incyte has announced that the FDA has approved MONJUVI (tafasitamab-cxix) in combination with rituximab and lenalidomide for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL). This makes it the f...
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Jun 18, 2025
Exploring Life-Saving Heart Devices: Innovations Transforming Cardiac Care
Heart disease continues to be a major global health concern, impacting countless lives and placing a significant burden on healthcare systems worldwide. According to the World Health Organization, cardiovascular diseases are responsible for approximately 17.9 million deaths each year, accounting for nearly one-thir...
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Jun 17, 2025
Celltrion Announces FDA Nod for New STEQEYMA Presentation to Broaden Pediatric Use; CSL’s ANDEMBRY Gets FDA Nod as First Factor XIIa Inhibitor for Hereditary Angioedema; Ocugen’s OCU410ST Cleared by FDA to Begin Phase II/III Trial in Stargardt Disease; Milestone Pharmaceuticals Submits FDA Response for CARDAMYST in PSVT; AbbVie’s MAVYRET Approved by FDA as First and Only Therapy for Acute Hepatitis C
Celltrion Announces FDA Approval of New STEQEYMA Presentation, Expanding Pediatric Dosing Options Celltrion, Inc. has announced that the FDA has approved an additional presentation of STEQEYMA (ustekinumab-stba), a biosimilar to STELARA, in a 45mg/0.5mL single-dose vial for subcutaneous injection. This new formu...
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Jun 10, 2025
Amylyx’s AMX0114 Fast Tracked by FDA for ALS; Cellectar’s Iopofosine I 131 Granted FDA Breakthrough in Waldenstrom Macroglobulinemia; YolTech’s YOLT-101 Clears FDA IND for Familial Hypercholesterolemia; Oncovita’s MVdeltaC Gets FDA Orphan Tag for Pleural Mesothelioma; Merck’s ENFLONSIA Approved for RSV Prevention in Infants
Amylyx Pharmaceuticals Receives FDA Fast Track Designation for AMX0114 in ALS Amylyx Pharmaceuticals announced that the FDA has granted Fast Track designation to its investigational antisense oligonucleotide (ASO), AMX0114, for the treatment of amyotrophic lateral sclerosis (ALS). The candidate targets calpain-2...
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Jun 04, 2025
Types of Mitochondrial Diseases and the Science Behind Them: A Journey from Dysfunction to Discovery
Mitochondrial diseases, affecting an estimated 1.5 million individuals globally (approximately 1 in 5,000), represent a complex group of rare genetic disorders driven by mitochondrial dysfunction, disrupting cellular energy production. These conditions stem from mutations in mitochondrial DNA (mtDNA) or nuclear DNA...
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