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With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding to the pressure, the company suffered a setback Monday when it halted two studies of Voxzogo in other growth-related conditions following multiple cases of hip injuries in other trials of the drug.
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While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”
After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question whether shared evidence and prior knowledge will accelerate development in rare diseases.
The U.S. Senate has a plan to improve drug development for rare disease patients. The exit of controversial CBER chief Vinay Prasad will help clear the path.
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Eli Lilly’s win in a head-to-head trial drove Novo Nordisk’s market cap to pre-Wegovy levels not long after the victor became the first pharma company to top a $1 trillion valuation. It seems one company can do no right, while the other can do no wrong.
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Among the unreported adverse events potentially linked to Ozempic are two deaths and one case of “completed suicide,” according to an FDA inspection report.
Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and outgoing Vinay Prasad, director of the Center for Biologics Evaluation and Research.
Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson launches an investigation into recent rare disease drug rejections; and Roche and Zealand’s amylin analog fails to match investor expectations—and Eli Lilly’s rival candidate—in a mid-stage trial.
Whether happening in public or private, biopharma M&A is fiercer than ever. Experts point to patent pressures, herd mentality and a declining stock of available biotechs with mature assets.
The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease gene therapy “bureaucratic idiocy.”
Industry and FDA representatives have reached a general agreement on planned pre-submission facility meetings but have expressed different views about the specifics.
The move comes as BioNTech shifts to being a multiproduct commercial biotech, allowing Ugur Sahin and Özlem Türeci to transition back into research on next-generation mRNA therapeutics.
Analysts expect the market for manufacturing cell and gene therapies, worth less than $20 billion in 2024, to expand rapidly as approvals drive higher volumes of production.
Breakout Ventures’ focus on early-stage companies stands out as more and more investors elect to save their dollars for derisked assets.
Stylus Medicine, a member of BioSpace’s NextGen Class of 2026, launched in May 2025 to develop new, less complex genetic medicines. The company’s in vivo approach has attracted “intense” interest from Big Pharma.