Stress led all triggers, and a sense of an impending attack was the most reliable prodrome in patients with HAE-C1INH in the US HAEA Scientific Registry.
Approval is based on 36-week data from BRAVE-AA-PEDS, in which 42% of patients on the 4 mg dose achieved a SALT score of 20 or less, compared with 5% on placebo.
The FDA approved once-daily oral zilurgisertib for FOP in patients aged 12 years and older after the phase 2 PROGRESS trial showed reduced new heterotopic ossification vs placebo.
As the FDA weighs its first psychedelic approval, Sarah Norman, MPP, says training, credentialing, and reimbursement are lagging while patients are already asking.