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 […]
CDC Advisors Endorse Maternal RSV Vax to Protect Newborns
The CDC’s advisors on Friday recommended a maternal respiratory syncytial virus (RSV) vaccine to protect infants from serious infections. By an 11-1 vote, the Advisory Committee on Immunization Practices (ACIP) recommended that pregnant women receive a single dose of Pfizer’s prefusion F protein (RSVpreF) vaccine (Abrysvo) at 32 to 36 weeks’ gestation to prevent lower […]
FDA Panel Reluctantly Backs Patisiran for ATTR Cardiomyopathy
FDA advisors conceded on Wednesday that the benefit-risk balance of patisiran (Onpattro) technically checks out in wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM, also known as cardiac ATTR amyloidosis) — though they were unconvinced that the benefits are actually meaningful. At the end of the day, the Cardiovascular and Renal Drugs Advisory Committee ultimately voted […]
Medtronic’s Renal Denervation System for Hypertension Splits FDA Panel
A radiofrequency-based renal denervation system for uncontrolled hypertension got a cool reception from FDA advisors who nevertheless left the door open for regulatory approval. On Wednesday, the FDA’s Circulatory System Devices Panel voted 6-7 (with one abstention) that the benefits of the Symplicity Spyral multi-electrode catheter system do not outweigh its risks when used to […]
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. […]
All Thumbs Up for OTC Birth Control Pill at FDA Advisory Meeting
In a unanimous vote, advisors to the FDA recommended the agency approve what could become the first-ever over the counter (OTC) birth control pill. All 17 members of the joint Nonprescription Drugs Advisory Committee and the Obstetrics, Reproductive, and Urologic Drugs Advisory Committee said the widespread benefits of switching daily norgestrel tablets 0.075 mg (Opill) […]
FDA Panel Rejects Broad Olaparib Indication in Metastatic CRPC
In a near-unanimous vote on Friday, outside advisors to the FDA recommended a narrow indication should the agency approve olaparib (Lynparza) plus abiraterone (Zytiga) as initial treatment for metastatic castration-resistant prostate cancer (mCRPC). In an 11-1 vote (with one abstention), the Oncologic Drugs Advisory Committee (ODAC) recommended that any approval be restricted to patients with […]
FDA Advisors Endorse Rexulti for Agitation in Alzheimer’s Dementia
FDA advisors voted in favor of brexpiprazole (Rexulti) for treating agitation associated with Alzheimer’s dementia (AAD), despite increased mortality risk. By a vote of 9-1 on Friday, members of the agency’s Psychopharmacologic Drugs Advisory Committee (PDAC) and the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) said the data presented by the drug’s sponsors, […]
FDA Panel Supports Tofersen for Rare Genetic ALS
A panel of FDA advisors voted unanimously that plasma neurofilament light chain (NfL), a biomarker of neurodegeneration, is a reasonable surrogate endpoint in amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene (SOD1-ALS). In a 9-0 vote on Wednesday, the agency’s Peripheral and Central Nervous System Drugs Advisory Committee said the available […]
Second RSV Vaccine for Older Adults Gets Thumbs Up From FDA Advisors
A panel of FDA’s outside advisors recommended the agency approve GSK’s respiratory syncytial virus (RSV) vaccine for older adults, a day after backing Pfizer’s RSV vaccine candidate for that same population. By votes of 12-0 on efficacy and 10-2 on safety, the Vaccines and Related Biological Products Advisory Committee (VRBPAC) said the available data support […]