Regulatory

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.

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.

CSL Vifor holds the marketing authorization for Tavneos in the U.K., where the drug is marketed as Avacopan Vifor. It continues to be sold on the U.S. market by Amgen despite concerns raised by the FDA.

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.

The U.S. is scrambling to compete with a strategically positioned China on the biotech stage as the Asian country surpasses America in biomedical research and clinical trials. So far, China is winning.

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.

In this episode of Denatured, you’ll hear from Hilary Rosselot, executive director at the National Fragile X Foundation and Jordi Fàbrega, co-founder & CEO at CONNECTA Therapeutics. From trial endpoints and placebo effects to neuroplasticity modulation, we explore what it will take to build a sustainable path to the first approved therapy.

The FDA granted the approval of Revolution Medicines’ daraxonrasib, now to be known as Rasonque, in pancreatic adenocarcinoma 6.5 months ahead of deadline.