The approval was based on the results from 3 studies evaluating the efficacy of Nulibry in patients with MoCD Type A vs data from natural history with results demonstrating a reduced risk of death (82%) & increased probability of survival (84%) @3yrs. vs 55% in the historical control group
The US FDA approved Nulibry in Feb’21 for reducing the mortality rate of patients with MoCD Type A whereas, following the CHMP recommendation, an application for the marketing approval for Nulibry in the EU is expected to be submitted in 2022
In Dec’19, Medison acquired the commercialization rights of Nulibry in Israel whereas Sentynl acquired the global rights for the development & commercialization of Nulibry in Mar’22. Sentynl & BridgeBio co-share the development responsibilities through MAA under accelerated assessment with the EMA
Ref: PR Newswire | Image: Bridgebio
