Update on Duvyzat™ (givinostat) for Duchenne Muscular Dystrophy
Duvyzat™ receives positive recommendation from the EMA’s CHMP for conditional approval in DMD.
Click to read what this could mean for families and what happens next.
EMA confirms recommendation not to renew marketing authorisation for Translarna
The CHMP agreed to re-examine its decision on request of the manufacturer PTC. This took place in January and despite efforts from the manufacturer and patient community the EMA announced on Friday, 26 January 2024 recommendation for non-renewal of authorisation of Translarna. The process found that while there was evidence of benefit from the treatment, it’s not statistically significant. Importantly there was no concerns about the safety of the treatment.