Targeting an ultrarare disease affecting about 250 U.S. residents, Immedica Pharma AB’s Loargys (pegzilarginase) won accelerated FDA approval to treat hyperargininemia in those ages 2 and older with arginase 1 deficiency (ARG1-D).
Japan is backing conditional approvals of Amchepry (raguneprocel, Sumitomo Pharma Co. Ltd./Racthera Co. Ltd.) and Reheart (Cuorips Inc.), positioning them to become the world’s first induced pluripotent stem cell-derived (iPSC) therapies to receive regulatory clearance.
The U.S. FDA accepted, with priority review, Takeda Pharmaceutical Co. Ltd.’s NDA submission for oveporexton (TAK-681), bringing the oral orexin receptor 2 agonist closer to clearance in narcolepsy type 1.
Tessera Therapeutics Inc.’s lead in vivo gene editing program, TSRA-196, has been awarded orphan drug and fast track designations by the FDA for adults with α-1 antitrypsin deficiency (AATD).
The ramifications of the U.S. Supreme Court’s decision Feb. 20 that shot down President Donald Trump’s reciprocal tariffs issued under the International Emergency Economic Powers Act are rippling across the world. And Trump’s immediate response to that ruling – a proclamation imposing a temporary 10% import duty on most goods brought into the country beginning Feb. 24 – isn’t helping.
Shares in Novo Nordisk A/S took another battering after the company announced its next-generation obesity drug Cagrisema failed to show noninferiority to Eli Lilly and Co. Inc.’s Zepbound in an open-label comparator study.
Amid an ongoing court challenge to the current composition of the CDC’s Advisory Committee on Immunization Practices (ACIP), the committee’s Feb. 25-27 meeting has been removed from its calendar.
The U.S. FDA rolled out its anticipated “plausible mechanism” draft guidance, enabling ultrarare disease drug developers to generate evidence of effectiveness and safety to support approval when randomized controlled trials are not possible due to small patient populations.
Vanda Pharmaceuticals Inc. gained U.S. FDA approval for Bysanti (milsaperidone), cleared for use in acute bipolar I disorder and schizophrenia and ensuring continuation of the firm’s atypical antipsychotic franchise, with Fanapt (iloperidone) set to start losing patent protection in 2027. It also offered a rare straightforward regulatory win for Vanda, which has tussled with the FDA in recent years.
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