fda

First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.

Vepdegestrant becomes the first PROTAC ever approved by the FDA, treating ESR1-mutated advanced breast cancer after one prior endocrine therapy.

The FDA announced two proof-of-concept real-time cancer trials and opened public comment on a broader pilot program, betting that cloud and AI-mediated data flow can compress drug review timelines.

A new joint program from FDA and CMS would issue a proposed Medicare coverage decision the same day a breakthrough device wins FDA authorization, aiming to cut total time-to-coverage from roughly a year or more to about two months.

Compass Pathways, Usona Institute, and Otsuka-acquired Transcend Therapeutics have been selected for FDA's Commissioner's National Priority Voucher program for psilocybin and methylone, six days after President Trump's executive order on serious mental illness.

Regeneron's one-time intracochlear gene therapy for OTOF-related deafness cleared the FDA 61 days after BLA filing, the first gene therapy product approved under the Commissioner's National Priority Voucher pilot program.

Axsome Therapeutics' AXS-05 faces its FDA verdict on April 30 for Alzheimer's disease agitation, backed by four Phase 3 trials but complicated by one failed study.

CorTec's implantable Brain Interchange system received FDA Breakthrough Device Designation on April 8 and TAP acceptance on April 23, marking the first BCI worldwide designated for stroke motor rehabilitation.

The FDA cleared doravirine/islatravir on April 21, completing a development program that was halted in 2021 over lymphocyte counts and restarted at a lower dose.

After FDA 510(k) clearance in December and a 45,000-patient global milestone in March, the Cambridge-based surgical robotics company begins placing its modular, lower-cost Versius Plus system in US hospitals.

Denali's tividenofusp alfa becomes the first FDA-approved biologic engineered to cross the blood-brain barrier, breaking a long rare-disease rejection streak and validating a transport-vehicle platform that could unlock CNS access for many more drugs.

Phase 3 data presented at AAN 2026 show azetukalner nearly halved seizure frequency in treatment-resistant focal epilepsy, with 38% of long-term patients achieving over a year of seizure freedom.