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 […]
Nursing Leaders Tackle Ways to Fix ‘Accelerated Burnout’ in New Nurses
Nursing leaders shared strategies for addressing “accelerated burnout” among nurses new to the profession during a panel discussion hosted by the National Council of State Boards of Nursing (NCSBN) on Thursday. Approximately 100,000 RNs left the workforce during the COVID-19 pandemic and another 800,000 anticipate leaving in the next 5 years, said Maryann Alexander, PhD, […]
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. […]
Drug for Rare Form of ALS Earns FDA Nod
The FDA granted accelerated approval to tofersen (Qalsody) for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, the agency announced on Tuesday. The drug was cleared under the accelerated approval pathway, a program that uses a surrogate endpoint to approve drugs for serious conditions with an unmet medical need. For tofersen, the […]
FDA Greenlights New First-Line Regimen in Bladder Cancer
The FDA gave accelerated approval to enfortumab vedotin (Padcev) and pembrolizumab (Keytruda) as first-line treatment for certain patients with locally advanced or metastatic urothelial cancer, the agency announced on Monday. Specifically, the combination is indicated for those who are ineligible for cisplatin-containing chemotherapy, and the approval marks the first anti-PD-1/antibody-drug conjugate combination for this patient […]
FDA OKs Sparsentan for IgA Nephropathy-Related Proteinuria
The FDA granted accelerated approval to sparsentan (Filspari) for proteinuria in IgA nephropathy, Travere Therapeutics announced, marking the first non-immunosuppressive therapy for this rare condition. Also known as Berger’s disease, proteinuria in IgA nephropathy is the leading cause of kidney failure due to glomerular disease, and sparsentan is specifically indicated to reduce proteinuria in adults […]
Michael Wang on the Reversible BTK Inhibitor for Previously Treated MCL
The FDA recently granted accelerated approval to pirtobrutinib (Jaypirca), a reversible Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of relapsed or refractory mantle cell lymphoma (MCL) after at least two prior lines of systemic therapy, including a BTK inhibitor. In this exclusive MedPage Today video, Michael Wang, MD, of the University of Texas MD […]
Accelerated Approval Not the Finish Line, Says FDA Commissioner
Drugmakers will no longer be able to slow-walk their accelerated approvals to full regulatory approval, said FDA Commissioner Robert Califf, MD, during the J.P. Morgan Healthcare Conference on Tuesday, while also fielding questions on gene therapies and the modernization of clinical trials, among other topics. Accelerated Approval When asked about the kind of data needed […]
Alzheimer’s Drug Lecanemab Wins FDA Approval
Lecanemab (Leqembi) was approved under the accelerated approval pathway to treat Alzheimer’s disease, the FDA announced on Friday. The anti-amyloid should be started only in patients with mild cognitive impairment or who are at the mild dementia stage of Alzheimer’s disease, the population studied in clinical trials, the agency noted, and the drug label indicates […]
FDA Broadens Options for Relapsed/Refractory Multiple Myeloma With Teclistamab
Treatment options for relapsed/refractory multiple myeloma continue to expand with FDA’s accelerated approval of teclistamab (Tecvayli). The approval stipulates use of the bispecific antibody in adults who have received at least four prior therapies, including an immunomodulator, a proteasome inhibitor, and an anti-CD38 antibody (triple-class refractory disease). The first approved drug of its kind, teclistamab […]