Regeneron wins FDA approval to challenge Ipsen for rare bone disease market
Regeneron’s Pasatru is the second fibrodysplasia ossificans progressiva drug approved in the U.S., following the 2023 approval of Ipsen’s Sohonos.
FDA News: Approvals, Adcomms, Complete Response Letters and Guidances
FDA News: Approvals, Adcomms, Complete Response Letters and Guidances
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Ultragenyx bags first gene therapy approval as previously rejected Sanfilippo asset awaits decision
The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September.
Why pediatric rare oncology outperforms in a volatile biotech market
Real progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investment, even in a capital-constrained environment.
Trump's FDA pick Overton good for psychedelics, new pain meds but bad for vaccines: Analysts
If confirmed by the Senate, Heidi Overton—officially revealed Wednesday as President Donald Trump’s choice for FDA commissioner—is expected to be a boon for developers of psychedelic therapeutics and non-opioid pain drugs, but could tow the party line on vaccines.
Trump picks White House aide Heidi Overton as FDA chief: Reports
If Heidi Overton clears the Senate nomination process, analysts say she will have a tough job ahead to “restore predictability” to an agency battered by layoffs and controversial policies.
Amylyx’s redemption story, Summit’s survival woes, Definium’s anxiety win, more
Amylyx shares good news for its investigational GLP-1 blocker; Definium announces positive data for its LSD-based therapy in anxiety; Summit/Akeso’s ivonescimab shows degrading survival benefits; patients treated with Neurocrine’s Prader-Willi drug experience serious adverse events; and Eli Lilly cracks down on the black market for next-gen obesity asset retatrutide.
Capricor soars on potential FDA review extension, filing amendments
Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.
BMS welcomes new multiple myeloma drug as generics batter current portfolio
The FDA approves Zenbexus just as copycat competitors depress sales of Bristol Myers Squibb’s multiple myeloma drugs Pomalyst and Revlimid, which crashed 71% and 49% year-on-year, respectively, in the second quarter.
Nascent US psychedelic therapy opportunity attracts overseas manufacturers
Companies that manufacture MDMA and psilocybin for patients in other countries are looking to use their experience to access the potential U.S. market.
Taiho and Cullinan’s zipalertinib clears Phase 3, teeing up first-line competition
Taiho Pharmaceutical and Cullinan Therapeutics’ cancer asset is already under FDA review in a later setting, with a decision expected by February.