The FDA granted accelerated approval to Viltepso (viltolarsen) to treat Duchenne muscular dystrophy patients (DMD) who have a confirmed mutation of the DMD gene amenable to exon 53 skipping, pitting it against Sarepta Therapeutics Inc.’s Vyondys (golodirsen), which was given accelerated approval last year in a controversial decision by the FDA following an agency complete response letter (CRL) highlighting safety concerns.
More than two weeks ahead of its expected PDUFA date, PTC Therapeutics Inc.’s spinal muscular atrophy (SM) drug, risdiplam, gained FDA approval, making it the first at-home, oral treatment intended for use in adults and children 2 months and older.
Two weeks after receiving a letter from the FDA citing deficiencies in the NDA seeking approval for VP-102 (cantharidin 0.7% topical solution) in molluscum contagiosum, Verrica Pharmaceuticals Inc. disclosed an unsurprising complete response letter.
A month ahead of its PDUFA date, Ultragenyx Pharmaceutical Inc.’s NDA for triheptanoin (UX-007) won FDA approval. Branded Dojolvi, the highly purified, synthetic, seven-carbon fatty acid triglyceride was cleared for use in treating pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
The U.S. District Court for the Northern District of West Virginia ruled against Biogen Inc., of Cambridge, Mass., and in favor of Mylan NV, of Hertfordshire, U.K., in a patent dispute regarding Biogen’s blockbuster multiple sclerosis drug, Tecfidera (dimethyl fumarate).
Shares of Gilead Sciences Inc. were dented April 23 after reports surfaced that its antiviral drug, remdesivir, failed to improve the condition of patients with COVID-19.
Shares of Gilead Sciences Inc. were dented April 23 after reports surfaced that its antiviral drug, remdesivir, failed to improve the condition of patients with COVID-19.
Two phase III studies in China testing Gilead Sciences Inc.’s antiviral drug, remdesivir, in patients with COVID-19 infection have been halted after Chinese authorities reported a lack of eligible patients. Other studies, including trials sponsored by the Foster City, Calif.-based company, remain ongoing.
Two phase III studies in China testing Gilead Sciences Inc.’s antiviral drug, remdesivir, in patients with COVID-19 infection have been halted after Chinese authorities reported a lack of eligible patients. Other studies, including trials sponsored by the Foster City, Calif.-based company, remain ongoing.
News of a partial FDA clinical hold for pediatric patients after safety reports from an ongoing trial testing palovarotene in fibrodysplasia ossificans progressiva (FOP) identified cases of early growth plate closure sent shares of Ipsen SA (Paris:IPN) falling 13.6% Friday.
