FDA Panel Votes Against Broad Talazoparib-Enzalutamide Label in Prostate Cancer
An FDA advisory committee on Wednesday unanimously voted against recommending approval of the PARP inhibitor talazoparib (Talzenna) plus enzalutamide (Xtandi) for all patients with metastatic castration-resistant prostate cancer (mCRPC). In an 8-0 vote, members of the Oncologic Drugs Advisory Committee (ODAC) said the available data from the TALAPRO-2 trial do not support a favorable risk-benefit […]
FDA Approves First-Ever Drug for Serious Liver Condition
The FDA granted accelerated approval to resmetirom (Rezdiffra) as the first treatment for adults with noncirrhotic non-alcoholic steatohepatitis (NASH), also now referred to as metabolic dysfunction-associated steatohepatitis (MASH). A once-daily oral agent, resmetirom is a liver-directed thyroid hormone receptor (THR)-β selective agonist designed to target key underlying causes of NASH; the drug is specifically indicated […]
Epinephrine Nasal Spray Starts Reversing Allergic Reactions in Minutes
WASHINGTON — Although FDA approval has eluded the intranasal epinephrine spray ARS-1 (also known as neffy), it continued to build its case with findings of resolved allergic symptoms in children and teenagers undergoing an oral food challenge, and with a separate study showing that it maintained its potency under extreme temperatures. In a small observational […]
Drug Candidate for Chronic Cough Rejected by FDA Panel
FDA advisors told the agency that the current evidence does not support approval of gefapixant for chronic cough, a condition where no approved therapy exists. On Friday, members of the Pulmonary-Allergy Drugs Advisory Committee voted 12-1 that the small reductions in cough frequency demonstrated in phase III trials of the P2X3 receptor antagonist do not […]
FDA Panel: Tolerating Approval Outliers Would Set ‘Dangerous Precedent’
An unprecedented wait for final FDA approval will drag on for two orphan lymphoma drugs, at least a little while longer, as the agency decides how to end the delays and prevent them from happening again. The dihydrofolate reductase inhibitor pralatrexate (Folotyn) received accelerated approval in 2009 and the histone deacetylase (HDAC) inhibitor belinostat (Beleodaq) […]
FDA OKs Another Injectable for Rare Kidney Disorder
Nedosiran (Rivfloza), a once-monthly RNA interference (RNAi) injection, gained FDA approval for certain patients with primary hyperoxaluria type 1 (PH1), drugmaker Novo Nordisk announced Monday. The lactate dehydrogenase A-directed small interfering RNA is indicated to lower urinary oxalate levels in patients ages 9 years and older with relatively preserved kidney function, defined as an eGFR […]
FDA Relents, Approves Novel Antidepressant After Many Rejections
After multiple prior rejections, gepirone hydrochloride extended-release (ER; Exxua) gained FDA approval for the treatment of major depressive disorder (MDD) in adults, drugmaker Fabre-Kramer Pharmaceuticals announced. Gepirone ER is the first approved antidepressant that selectively targets the serotonin 1A receptor, which regulates mood and emotion. The drug was approved based on data from two 8-week […]
FDA Approves First Oral Antiviral Drug for COVID-19
The FDA has granted full approval to nirmatrelvir-ritonavir (Paxlovid) for treating adult outpatients with mild to moderate COVID-19 who are at risk for severe disease, the agency announced on Thursday. “Today’s approval demonstrates that Paxlovid has met the agency’s rigorous standards for safety and effectiveness, and that it remains an important treatment option for people […]
Rare Skin Blistering Condition Gets First Drug Approved
The FDA granted the first drug approval for dystrophic epidermolysis bullosa (DEB), a topical gene therapy that treats the blistering wounds it causes, the agency announced Friday. The herpes-simplex virus type 1 vector-based gene therapy, Vyjuvek, was approved for patients ages 6 months and older with mutation in the COL7A1 (collagen type VII alpha 1 […]
Duchenne Muscular Dystrophy Treatment Narrowly Captures Support of FDA Advisors
FDA advisors narrowly supported accelerated approval for Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD) on Friday. In a 8-to-6 vote, the agency’s Cellular, Tissue, and Gene Therapies Advisory Committee said the overall benefits and risks supported SRP-9001 (delandistrogene moxeparvovec) for ambulatory Duchenne patients, using expression of Sarepta’s micro-dystrophin as a surrogate endpoint. […]