FDA
Dec 24, 2024
FDA Approves Vertex’s ALYFTREK for Cystic Fibrosis; ZEPBOUND Gets FDA Approval for Obstructive Sleep Apnea in Obese Adults; Tonix’s TNX-102 SL NDA Accepted for Fibromyalgia; Quoin’s QRX003 Clears FDA for Netherton Syndrome Study; FDA Approves First Mesenchymal Stromal Cell Therapy for Acute GvHD
Vertex Receives US FDA Approval for ALYFTREK: A Breakthrough CFTR Modulator for Cystic Fibrosis Vertex Pharmaceuticals Incorporated announced that the FDA had approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), a once-daily triple combination CFTR modulator for the treatment of cystic fibrosis. ALYFTREK is...
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Dec 17, 2024
FDA Approves Neurocrine’s CRENESSITY for Congenital Adrenal Hyperplasia; Checkpoint’s UNLOXCYT Approved; MAIA’s THIO Gets Rare Pediatric Disease Designation; JEMPERLI Receives Breakthrough Therapy for Rectal Cancer; Galderma’s NEMLUVIO Approved for Atopic Dermatitis.
Neurocrine Announces FDA Approval of CRENESSITY for Congenital Adrenal Hyperplasia Neurocrine Biosciences, Inc. announced that the FDA has approved CRENESSITY™ (crinecerfont) as an adjunctive treatment to glucocorticoid replacement for controlling androgens in adult and pediatric patients aged four years and old...
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Dec 10, 2024
AbbVie Reveals Phase III TEMPO-2 Trial Positive Topline Results; FDA Accepts GSK’s NUCALA Submission in COPD; Novartis Boosts Huntington’s Disease Development Program with PTC518 In-Licensing; Chimerix to File for Accelerated FDA Approval of Dordaviprone for H3 K27M-Mutant Diffuse Glioma; MeiraGTx Receives FDA RMAT Designation for AAV2-hAQP1 in Grade 2/3 Radiation-Induced Xerostomia Treatment
AbbVie Unveils Positive Phase III TEMPO-2 Trial Findings for Tavapadon as Parkinson's Disease Monotherapy AbbVie announced favorable topline results from its pivotal Phase III TEMPO-2 trial, which assessed the investigational drug tavapadon as a flexible-dose monotherapy for early-stage Parkinson's disease. Tava...
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Dec 03, 2024
FDA Grants Fast Track for Lin BioScience’s LBS-007; Alnylam’s AMVUTTRA sNDA Under Review; FDA Approves RAPIBLYK for Atrial Fibrillation; Applied Therapeutics Receives CRL for Govorestat in Galactosemia; R289 Gets Fast Track for Lower-Risk MDS.
Lin BioScience Receives FDA Fast Track Designation for LBS-007 Lin BioScience, a clinical-stage biopharmaceutical company focused on developing innovative therapies for acute leukemia, announced that its lead pipeline candidate, LBS-007, has been granted Fast Track Designation by the FDA for the treatment of acu...
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Nov 26, 2024
FDA Grants Orphan Drug Designation for Ractigen’s RAG-21 in ALS; Intellia’s Nexiguran RMAT for ATTR Amyloidosis; FDA Approves Acoramidis for ATTR-CM; Jazz Wins FDA Approval for Ziihera in HER2-Positive Biliary Cancer; J&J Seeks FDA Approval for Subcutaneous TREMFYA in Colitis
Ractigen Therapeutics Receives FDA Orphan Drug Designation for RAG-21 to Treat ALS Ractigen Therapeutics has announced that the FDA has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy for treating amyotrophic lateral sclerosis (ALS). RAG-21 specifically targets FUS-ALS, one of the most a...
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Nov 19, 2024
FDA Approves PTC’s AADC Gene Therapy; DUPIXENT sBLA Acceptance for Urticaria; CHMP Recommends TAGRISSO for EGFR Lung Cancer; FDA Approves DANZITEN for CML; Syndax Wins FDA Approval for REVUFORJ in Acute Leukemia
FDA Approves AADC Deficiency Gene Therapy by PTC Therapeutics PTC Therapeutics, Inc. has secured FDA accelerated approval for KEBILIDI (eladocagene exuparvovec-tneq), marking a historic milestone as the first-ever gene therapy directly administered to the brain in the United States. This innovative therapy addre...
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Nov 12, 2024
AUCATZYL Approved for R/R B-ALL; FDA Accepts NDA for Unicycive’s Oxylanthanum Carbonate; AstraZeneca and Amgen Report Positive Results in Chronic Rhinosinusitis; Nipocalimab Granted Breakthrough Designation for Sjögren’s Disease; AbbVie’s Schizophrenia Drug Fails Phase Studies
FDA Approves Autolus's AUCATZYL for Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia Autolus Therapeutics has achieved a significant milestone with FDA approval for AUCATZYL (obecabtagene autoleucel), a next-generation CAR T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic le...
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Nov 05, 2024
Prolong’s PP-007 Fast-Tracked for Stroke; FDA Expands JYLAMVO Pediatric Approval; Corcept’s Cushing’s Drug Shows Positive Phase III Results; ESSA Halts Phase II Study of Masofaniten for Prostate Cancer; SCEMBLIX Approved for Leukemia
Prolong Pharmaceuticals Secures FDA Fast Track for PP-007 in Stroke Therapy Prolong Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company, announced that its investigational therapy, PP-007 (PEGylated carboxyhemoglobin, bovine), has been granted Fast Track designation by the FDA for the treatment of a...
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Nov 01, 2024
5 Promising Pompe Disease Drugs in Early-stage Development
Imagine a disease that stealthily incapacitates the heart, liver, and skeletal muscles—slowly breaking them down due to the absence of a crucial protein. That's the story of Pompe disease, also known as glycogen storage disease type II (GSDII). Caused by a gene mutation that blocks the body’s ability to break down ...
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Oct 29, 2024
FDA Grants Orphan Status to MDL-101 for LAMA2-CMD; Pfizer’s ABRYSVO Approved for High-Risk Adults (18-59); KIND’s AND017 Gains Orphan Designation for Sickle Cell Disease; HiberCell’s HC-7366 Fast-Tracked for AML; ORLYNVAH Approved for Uncomplicated UTIs
FDA Grants Orphan Drug Status to MDL-101 for LAMA2-CMD Treatment Modalis Therapeutics Corporation has received Orphan Drug Designation (ODD) from the FDA for MDL-101, its promising treatment for congenital muscular dystrophy type 1A (LAMA2-CMD). This designation is a significant step, aimed at fostering the deve...
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