The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease.
BioWorld tracked 215 clinical trial readouts spanning phases I through III in December 2025, a sizeable increase from 170 updates in November, but below the 252 reported in October.
Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
Shares of San Diego-based Capricor Therapeutics Inc. (NASDAQ: CAPR) closed Dec. 3 at $29.96, up $23.60, or 371%, as investors cheered top-line data from the pivotal phase III Hope-3 trial testing cell therapy deramiocel in Duchenne muscular dystrophy.
The winding regulatory road for the BLA to Capricor Therapeutics Inc.’s cell therapy for Duchenne muscular dystrophy has more clarity. Out of a recent type A meeting between Capricor and the U.S. FDA, prompted by a complete response letter in July regarding lead asset CAP-1002 (deramiocel), the two are in agreement about a path to potential approval.
After July’s first-ever release of more than 200 complete response letters (CRLs) by the U.S. FDA, the agency now says it will release letters shortly after sponsors receive them. In addition, the FDA released a new batch of 89 CRLs from 2024 to now that are tied to pending or withdrawn applications.
After about a two-week absence as the U.S. FDA’s CBER director, Vinay Prasad’s return overall prompted a mild reaction on Wall Street for some stocks tied to companies developing cell and gene therapies.
The news that Vinay Prasad has stepped down as CBER director at the U.S. FDA had some biotech stocks literally jumping in joy as the market opened July 30. Meanwhile, Prasad’s decisions regarding vaccine development, as well as actions by Makary and HHS Secretary Robert Kennedy, are coming under fire.
The realignment within the U.S. FDA continued with reports of the removal of two high level executives. When asked by BioWorld if the Center for Biologics Evaluation and Research’s (CBER) Office of Therapeutic Products director and deputy director had been forced out and if so, why, an HHS spokesperson responded on background with a single sentence: “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science.”
The appointment May 6 of Vinay Prasad as the head of the U.S. FDA’s Center for Biologics Evaluation and Research (CBER) “bodes poorly” for Sarepta Therapeutics Inc.’s development-stage pipeline, said Wainwright analyst Mitchell Kapoor – and Wall Street reflected as much, as the stock (NASDAQ:SRPT) ended that day down 26.6% vs. an XBI drop of 6.6% – this ahead of the after-hours earnings disclosure that pushed the Cambridge, Mass.-based firm down even farther by more than another 20%, with the XBI unchanged.
