PTC Therapeutics Inc. is already established as a player in rare diseases, working with Roche Holding AG to develop and market Evrysdi (risdiplam) to treat certain patients with spinal muscular atrophy. With Evrysdi now approved in the U.S. and Europe, and Translarna (ataluren) approved in Europe for Duchenne muscular dystrophy, South Plainfield, N.J.-based PTC is approaching a crucial juncture with its first gene therapy product.
Given the FDA’s Vaccines and Related Biological Products Advisory Committee’s unanimous vote last month to recommend use of a booster dose of the Pfizer Inc.-Biontech SE COVID-19 vaccine in certain high-risk groups, it came as no surprise when the committee again voted unanimously Oct. 14 for a mirror use of Moderna Inc.’s proposed booster.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Adocia, Algernon, Asieris, Beigene, Blade, Cantargia, Eli Lilly, Homology, Kadimastem, Lundbeck, Merck, Otsuka, Pharmather, Regeneron, Revance, Roche, Sanofi, Sorrento, Viridian.
The FDA’s device center has posted a draft guidance that addresses unique device identifier (UDI) code requirements applied to low-risk products. The select update offers enforcement discretion in some instances for class I consumer health products, a switch partly justified by the exceedingly low risk presented by such products.
Device makers have argued for years that not all medical device recalls are the same, and thus the FDA should be more forthcoming with the public about the difference between a recall that is accompanied by a market withdrawal and a recall that driven by something as innocuous as a minor adjustment to the product label.
Alpha Tau Ltd. has secured a second breakthrough device designation for its Alpha Dart radiation treatment for solid cancer tumors. The FDA has granted the Jerusalem-based company’s technology a designation for the treatment of patients with recurrent glioblastoma multiforme (GBM), an aggressive malignant brain tumor. GBM has an average five-year survival rate of less than 10% and is the most common malignant tumor of the brain or central nervous system. According to the designation, the Alpha Dart system can be used to treat recurrent GBM as an adjunct to standard medical therapies or as a standalone therapy after standard medical therapies have been exhausted.
Should Johnson & Johnson’s (J&J) COVID-19 vaccine be a two-dose series? While not directly asked, that question almost lurks between the lines of the FDA’s briefing document for the Oct. 15 meeting of its Vaccines and Related Biological Products Advisory Committee. The document referred to J&J’s proposed second dose as a “booster,” but the FDA isn’t asking the committee the questions it posed for the Moderna Inc. and Pfizer Inc.-Biontech SE boosters. Instead, it is inviting VRBPAC to advise on whether the second J&J dose should be administered two months or six months following the first shot.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Corium, Curevac, Deciphera, Gensight, Hoth, Intelgenx, Nrx, Ocular, Protara, Sofie, Viiv.