What a difference a U.S. FDA advisory committee meeting can make. In the wake of the Peripheral and Central Nervous System Drugs Advisory Committee voting 7-2 Sept. 7 to recommend approval of Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) candidate, shares of the Cambridge, Mass.-based company (NASDAQ:AMLX) more than regained the value they lost in March when the same committee voted against approval of AMX-0035.
The U.S. FDA’s approval of Revance Therapeutics Inc.’s Daxxify (daxibotulinumtoxinA-lanm) for the temporary improvement of moderate to severe glabellar lines, or frown lines, in adults, positioned the drug to compete with Abbvie Inc.’s blockbuster, Botox (onabotulinumtoxinA), which tallied $678 million in global net revenues for the second quarter of 2022.
The FDA has granted orphan drug designation to Iecure Inc.'s lead product candidate GTP-506, an investigational product for the treatment of ornithine transcarbamylase (OTC) deficiency.
Tenaya Therapeutics Inc. has received FDA clearance of its IND application to begin clinical testing of TN-301, a highly selective small-molecule inhibitor of histone deacetylase 6 (HDAC6) initially being developed for heart failure with preserved ejection fraction (HFpEF).
Monte Rosa Therapeutics Inc. has received FDA clearance of its IND application for MRT-2359, a potent and selective GSPT1-directed molecular glue degrader (MGD).
Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis candidate, AMX-0035, will get a rare second bite at the adcom apple Sept. 7. This time around, the Cambridge, Mass.-based company is looking to improve on its first performance by stressing the survival benefit of its drug.
Immuneering Corp. has submitted an IND application to the FDA to support a phase I/IIa trial of IMM-1-104, an oral once-daily small molecule in development for the treatment of advanced RAS-mutant solid tumors.
The U.S. FDA has become the first global regulator to approve Boehringer Ingelheim GmbH’s spesolimab, the first treatment specifically approved for generalized pustular psoriasis flares in adults, a rare and potentially fatal disease. Branded as Spevigo, it works by inhibiting interleukin-36 and is delivered via intravenous injection. Ingelheim, Germany-based Boehringer Ingelheim is not giving away details about pricing but Carinne Brouillon, a member of the company’s board responsible for human pharma, said Spevigo is “priced similarly to the other biologic therapies used to treat rare dermatologic diseases.”
The U.S. FDA’s formation of the Digital Health Center of Excellence was heralded as a key enabler of digital health technologies, but the news hasn’t necessarily had the expected effect. The agency’s December 2021 draft guidance on the use of digital health technologies to assist in the conduct of clinical trials is still in regulatory drydock.
The FDA has cleared Immusoft Corp.'s IND for ISP-001 (iduronicrin genleukocel-T) for the treatment of mucopolysaccharidosis type I (MPS I), allowing initiation of a phase I trial.
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