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 […]
FDA Grants De Novo Request for Neuromodulatory System for Urge Incontinence
The FDA has granted a de novo marketing request for the Revi System for tibial neuromodulation to treat urinary incontinence with or without urinary urgency, BlueWind Medical announced on Thursday. The de novo request allows for treatment of men and women with urge urinary incontinence (UUI). Clinicians should follow applicable clinical guidelines and use professional […]
FDA OKs Over-the-Counter Gel for Erectile Dysfunction
The FDA has granted over-the-counter marketing authorization to MED3000 (Eroxon), the first nonprescription topical gel for treating erectile dysfunction (ED), maker Futura Medical announced on Monday. According to the company, MED3000 has “a 10-minute onset of action” that sets it apart from prescription PDE5 inhibitors such as sildenafil (Viagra) and tadalafil (Cialis), which typically require […]
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 […]
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 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 […]
California’s Medical Misinformation Law Facing Legal Challenges
Late last month, a federal judge in California granted a preliminary injunction in a pair of related lawsuits filed in opposition to the state’s new medical misinformation law, which took effect January 1. The move temporarily blocks enforcement of the law for individual physicians who are plaintiffs in the suits as well as physicians who […]
Gene Therapy for Degenerative Brain Disorder Wins FDA Nod
The FDA granted accelerated approval to elivaldogene autotemcel (eli-cel; Skysona), a gene therapy to treat early, active cerebral adrenoleukodystrophy (CALD) in boys ages 4 to 17 years, drugmaker bluebird bio announced Friday. Childhood CALD is a rare, neurologically debilitating form of adrenoleukodystrophy that generally occurs in young boys. It is caused by a mutation in […]