U.S. Food and Drug Administration has approved Regeneron's gene therapy for a rare genetic form of deafness, the company said on Thursday.
Approval in severe-to-profound and profound OTOF -related hearing loss is based on pivotal results of the CHORD trial demonstrating 80% of participants achieved or surpassed a hearing level meeting the primary endpoint, and with longer follow-up, 42% achieved normal hearing that included whispers
Approval for children aged 2 to 11 years with CSU who remain symptomatic despite H1 antihistamine treatment based primarily on data from the LIBERTY-CUPID clinical trial program
As published in The Lancet and presented at AAN, NIMBLE trial met its primary and key secondary endpoints at week 24, demonstrating potential best-in-class efficacy and convenience in gMG