Pfizer’s gene therapy for a muscle-wasting disorder misses main goal of late-stage study

By Thomson Reuters Jun 12, 2024 | 3:42 PM

(Reuters) – Pfizer said on Wednesday its experimental gene therapy for a type of muscle-wasting disorder did not meet the main goal of a late-stage trial.

The study’s main goal was improvement in motor function among boys aged 4 to 7 years suffering from Duchenne muscular dystrophy (DMD) who can walk.

The therapy fordadistrogene movaparvovec also did not show significant difference compared to the placebo in key secondary goals.

Last month, the drugmaker said a young patient had died due to cardiac arrest after receiving the therapy in a mid-stage trial.

(Reporting by Puyaan Singh in Bengaluru; Editing by Vijay Kishore)