Following two complete response letters in the past year, Replimune Group Inc.’s RP-1 (suvolimogene oderparepvec) for unresectable metastatic cutaneous melanoma refractory to PD-1 therapy cleared a major hurdle when the U.S. FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 10-3 that the efficacy results from the Ignyte phase II study are evaluable and clinically meaningful.
The saga of Capricor Therapeutics Inc.’s allogeneic cell therapy deramiocel for cardiomyopathy in Duchenne muscular dystrophy (DMD) added another bleak chapter as the U.S. FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted on the question of whether the available data provide “substantial evidence” that the drug works. Balloting turned out 9 no, 3 yes.
Global drug approvals through the first half (H1) of 2026 reached 261, the third-highest H1 total in BioWorld’s records behind 2021’s 288 and 2025's 271, and ahead of 2022’s 256. June was the standout month of the first six months at 64 approvals, the highest single month in BioWorld’s records and a notable acceleration from May’s 43.
Kolon Tissuegene Inc.’s TG-C, an allogeneic cell and gene therapy candidate for knee osteoarthritis (OA), missed both co-primary endpoints and all key secondary endpoints in a highly anticipated top-line phase III clinical trial readout.
Otsuka Pharmaceutical Co. Ltd. will likely look next to expand the label of Simtriyo (centanafadine) into patients with comorbid anxiety, having won the U.S. FDA’s green light as a once-daily extended-release capsule for attention deficit hyperactivity disorder (ADHD) in adults plus pediatrics age 6 years and older who weigh at least 20 kg (about 44 lbs.).
The fight to keep Tavneos (avacopan) on the market is continuing, with Amgen Inc. submitting its defense of the vasculitis drug to the U.S. FDA just as the July 29 deadline approached. The submission is in support of a request for a hearing on Tavneos after the FDA proposed on April 30 that the marketing approval be annulled.
Suggesting Replimune Group Inc.’s statistical analysis method inflated responses in the single-arm phase II Ignyte trial of RP-1 (suvolimogene oderparepvec), briefing documents ahead of the U.S. FDA’s Cellular, Tissue, and Gene Therapies advisory committee meeting on July 30 sent the company’s shares spiraling downward by about 38%.
Dyne Therapeutics Inc. has obtained IND clearance from the FDA for DYNE-302 in facioscapulohumeral muscular dystrophy (FSHD), a rare, genetic disease caused by a mutation in the DUX4 gene. Dyne plans to conduct a phase I trial in ambulatory adults with FSHD.
Otsuka Pharmaceutical Co. Ltd. will likely look next to expand the label of Simtriyo (centanafadine) into patients with comorbid anxiety, having won the U.S. FDA’s green light as a once-daily extended-release capsule for attention deficit hyperactivity disorder (ADHD) in adults plus pediatrics age 6 years and older who weigh at least 20 kg (about 44 lbs.).
With its stock price falling as much as 70% July 27, Capricor Therapeutics Inc. took the rare step of issuing a news release to explain the negative tone of the U.S. FDA’s briefing document for the July 29 Cellular, Tissue and Gene Therapies Advisory Committee meeting to discuss the company’s BLA for deramiocel to treat cardiomyopathy in Duchenne muscular dystrophy.
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