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The approval of Mirum Pharmaceuticals’ ALK2 inhibitor for fibrodysplasia ossificans progressiva on Friday adds a third option for patients who had long been without any treatments, joining Regeneron’s recently approved Pasatru and Ipsen’s Sohonos.
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The recent FDA approval of Fayuvi for a rare, childhood neurodegenerative disease was “particularly special” for Ultragenyx CEO Emil Kakkis. It could also, along with an August nod for another gene therapy, help offset recent challenges brought on by two asset failures.
Johnson & Johnson, Pfizer, Argenx and more are unlocking new immunology targets and looking to improve on existing ones in an attempt to catch some of the magic brought about by AbbVie’s Humira and Sanofi and Regeneron’s Dupixent.
In Thursday’s Senate committee meeting, FDA commissioner nominee Heidi Overton was repeatedly questioned about her ability to retain the FDA’s independence under President Donald Trump, as well as about her views on vaccines and abortion drugs.
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Big Pharma needs more—not fewer—bold bets on rare and difficult diseases after Novartis’ efforts to invest in muscular dystrophy and ALS hit major roadblocks this month.
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Manufacturers are redesigning their processes to balance both better future emergency vaccine mobilization and the more demanding work of maintaining flexible, commercially viable manufacturing networks.
Nektar Therapeutics has been awarded $90 million in damages after a jury sided with the biotech, which had accused Big Pharma Eli Lilly of “botching” an analysis of their formerly partnered autoimmune drug.
Two RNA biotechs, ADARx Pharmaceuticals and City Therapeutics, are taking advantage of the IPO frenzy, which has led to a total of 27 newly public biotechs so far this year.
Ten patients out of 20 who received Johnson & Johnson and Legend Biotech’s Carvykti remained alive at five years of follow up.
Genentech’s agreement with Earendil Labs came the same day its parent company, Roche, inked a cardiometabolic and renal deal with Atavistik Bio for up to $2 billion.
FDA inspectors found evidence that the compounding pharmacy was making versions of Eli Lilly’s tirzepatide and Novo’s semaglutide “regularly or in inordinate amounts” at a site with “insanitary conditions.”
Analysts were keen to see a new modality compete with Roche’s approved drug Lucentis in the late-stage trial but new safety issues could complicate the future for Merck’s candidate.
Kyverna Therapeutics’ miv-cel showed improved mobility and a favorable safety profile at one year in patients with stiff person syndrome, further derisking an upcoming FDA application, William Blair said on Thursday. The biotech plans to file for priority review this year.
The weekly insulin Onswik will now be available to U.S. patients after initial approvals in Europe, Mexico and Japan.
Basecamp Research joins the growing group of AI-forward biotechs that in recent months have brought in notable sums from investors, including Isomorphic Labs and Enveda.