The U.S. Food and Drug Administration (FDA) has made several significant decisions affecting the biopharmaceutical industry this week.
UniQure announced that the FDA will require a randomized, double-blind, sham surgery–controlled Phase 3 trial for its gene therapy candidate targeting Huntington’s disease. This follows comments from FDA Commissioner Marty Makary that appeared to question the candidate's prospects.
REGENXBIO received a complete response letter from the FDA regarding its gene therapy for Hunter syndrome. The agency cited concerns about the study’s patient population, control group design, and reliance on surrogate markers to demonstrate efficacy. REGENXBIO had previously disclosed this setback in early February.
Regulatory specialists have voiced apprehension over what they describe as a narrowing “circle of trust” within the FDA. Some have characterized recent decisions as resembling “fiat,” impacting both individual drug reviews and broader policy changes. Reports indicate that the FDA has started an investigation into complaints about workplace culture under CBER director Vinay Prasad, who is closely involved in many regulatory calls.
Industry observers are also assessing the implications of the FDA’s shift from requiring two pivotal clinical trials for drug approvals to potentially accepting just one. Companies are seeking clarification on why this change is happening now, what risks it may introduce, and what evidence will be needed from a single trial.
In corporate developments, Sarepta Therapeutics CEO Doug Ingram resigned last week to focus on family matters as the company continues its efforts in muscular dystrophy treatments. During Sarepta’s fourth quarter earnings call, executives said sales of Elevidys—their Duchenne muscular dystrophy gene therapy—are expected to remain flat or decrease by up to 15% in 2026.
Eli Lilly outperformed Novo Nordisk again in an obesity treatment trial involving patients with type 2 diabetes, according to results from a Lilly-sponsored study. Novo Nordisk remains active in expanding its obesity pipeline; Tamara Darsow, a business development executive at Novo Nordisk, stated that the company is seeking new assets in this area. Last week, Novo announced a collaboration with Boston-based Vivtex to develop novel weight loss pills.
Additionally, BioPham Executive highlighted Merck’s Keytruda as retaining its status as the top-selling pharmaceutical product for 2025. The publication also reported that former 2seventy executive Chip Baird has been appointed CEO of Poplar Therapeutics—a newly launched firm that recently closed a $45 million series A extension funding round.
