FDA
The FDA aims to leverage the expertise of qualified research institutions paired with drug sponsors to lessen the risk of clinical holds during the investigational new drug review process and expedite the path to first-in-human development in the U.S. The initial application period will close on Oct. 30.
FEATURED STORIES
The FDA’s proposal to offer 50% off application fees for drugs with early-stage trials conducted in the U.S. is unlikely to achieve the intended result. What biotechs really want is speed.
Zanvastro, an antisense oligonucleotide therapy, represents the first independent neurology launch for Ionis Pharmaceuticals, which intends to leverage the foothold for more approvals in rare neurodegenerative diseases, including Angelman syndrome.
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.
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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.
September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.
The decision to promote Michael Davis and Karim Mikhail from acting to permanent division heads comes as the FDA awaits the potential confirmation of Heidi Overton, President Donald Trump’s nominee for commissioner.
The approval opens up a “modest” market for AstraZeneca’s Etcamah, according to Leerink analysts, who nevertheless looked forward to an upcoming late-stage readout that could lead to a “significantly larger opportunity” for the drug.
The FDA’s primary review team found that Replimune hadn’t sufficiently addressed deficiencies that had been pointed out in the two prior rejection letters for the melanoma therapy Tudriqev.
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.
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.