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.
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.
Although the need for COVID-19 boosters remains a tense debate among policymakers and scientific experts worldwide, the U.S. FDA is basing its Oct. 14-15 Vaccines and Related Biologics Products Advisory Committee meeting on the premise that vaccine boosters are needed.
FDA preemption of state liability law has proven controversial on a number of occasions, a fact of life resurrected by a case arising out of the Supreme Court of the State of Mississippi. The court declared that the FDA must invoke the rulemaking process for its regulation of medical product labels.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Athenex, Brii, Enzyvant, Genentech, Kintor, Merck, Protagonist, Regeneron, Ridgeback, Roche.
Shares of Protagonist Therapeutics Inc. (NASDAQ:PTGX) climbed 93.9% on Oct. 11 after the FDA removed a full clinical hold on studies of the rusfertide, the company's investigational treatment for the blood disorders polycythemia vera and hereditary hemochromatosis. Triggered by a finding of malignant skin tumors in mice treated with the drug disclosed on Sept. 17, the FDA's three-weeks-ago hold had pushed Protagonist shares down by as much as 72%.
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