FDA
The enrollment pause comes after BHV-7000 failed to show efficacy in a midstage depression trial late last year. The news is another regulatory hit for Biohaven after the FDA declined to approve its spinocerebellar ataxia treatment in November 2025.
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Last month’s FDA advisory committee meetings for therapies developed by Capricor Therapeutics and Replimune effectively illustrate the different outcomes for patients when the agency prioritizes specialization in its reviews.
Inspection access is not “an administrative footnote,” leaders of key FDA divisions wrote in an editorial on Wednesday vowing to expand oversight of foreign clinical trial sites.
Following a tumultuous stretch under ex-Commissioner Marty Makary, current FDA leadership is working to restore reliability, integrity and transparency to the agency—but experts say that doesn’t necessarily mean an easier regulatory road for drugmakers.
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The biologics license application follows months of public speculation—and regulatory reversals—for uniQure’s AMT-130. If accepted by the FDA for priority review, approval could come in the second quarter of 2027.
Novartis hits pause on several CAR T trials after three patient deaths, followed by Bristol Myers Squibb after separate safety signals; Revolution Medicines scores early FDA approval for groundbreaking pancreatic cancer drug; BioNTech suspends a mid-stage trial of its mRNA cancer immunotherapy after a futility analysis; and nine mid-sized biopharma companies join their big pharma peers in President Donald Trump’s Most Favored Nation drug pricing scheme.
The approval of rusfertide—now to be branded Mimrylo—for polycythemia vera marks a key win for Takeda, which now has crossed the FDA finish line for two of three critical product launches slated for the coming year.
With the approval, Eli Lilly’s Mounjaro becomes the first dual GIP/GLP-1 receptor agonist indicated to lower the risk of heart attack, stroke or cardiovascular death in adults with type 2 diabetes.
Lisraya carries a boxed warning typical of JAK inhibitors, but otherwise has a broad label that allows use in all adults with the rare autoimmune disease, setting up the drug for blockbuster status in a few years, according to Leerink.
The FDA granted the approval of Revolution Medicines’ daraxonrasib, now to be known as Rasonque, in pancreatic adenocarcinoma 6.5 months ahead of deadline.
Gastroesophageal adenocarcinoma presents the biggest market opportunity for Jazz Pharmaceuticals’ Ziihera, according to Truist Securities, which forecasts peak sales of the drug to reach $2.5 billion.
Though the potential change is unconfirmed, media outlets reported Wednesday that one deputy commissioner would focus on the intersection between health and AI, while another will be dedicated to drugs.
FDA extends its review of Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy as an unhappy shareholder advocates for change; Merck and Moderna tout positive data for their mRNA-based cancer vaccine; Trump nominates White House aide Heidi Overton as FDA commissioner; and the oral obesity market heats up with rivals Eli Lilly and Novo Nordisk going head-to-head.
The FDA has granted a three-month extension for Capricor Therapeutics’ Duchenne muscular dystrophy hopeful deramiocel in order to review additional data and consider a refined indication. The new target action date for the therapy is November 22.