The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) recommended expanding the use of Translarna (ataluren) to ambulatory children ages 2-5 with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD).
CHMP’s recommendation of Translarna use in younger children is based on PTC Therapeutics’ analysis of Phase 2 trial (NCT02819557) results which confirmed a positive benefit-risk ratio for these patients, according to CHMP.
The committee has also suggested the renewal of the current marketing authorization of Translarna.
“Early diagnosis and treatment has been a paramount part of our strategy and this recommendation perfectly aligns with our vision of giving best-in-class treatment to patients,” Marcio Souza, chief operating officer of PTC, said in a press release.
The multiple-dose, open-label Phase 2 trial conducted by PTC investigated the safety and tolerability profiles of Translarna in kids ages 2-5 with DMD by a “nonsense mutation” in the dystrophin gene.
This type of genetic mutation leads to the production of unfinished and nonfunctional dystrophin protein.
Researchers measured the number of adverse events as a primary safety measure and changes in body weight, BMI, time to walk/run 10 meters, maximum plasma concentration, and other parameters as secondary outcome measures.
Now, PTC announced positive results from this Phase 2 trial, leading to CHMP’s recommendation to expand the indication of the medicine to younger DMD children.
This decision comes after the U.S. Food and Drug Administration rejected Translarna last year for the treatment of DMD caused by nonsense mutations.
In September 2017, the FDA advisory panel concluded that there wasn’t enough evidence supporting Translarna’s effectiveness for this indication, and more research was needed.
“Ultimately, no positive results from any prospectively planned analyses that are persuasive have been provided with this application,” the agency stated.
In Europe, Translarna is conditionally approved by the EMA for the treatment of DMD patients 5 and older. The CHMP recommendation would expand the use of Translarna to children as young as 2.
In anticipation of the CHMP recommendation, PTC intensified its focus on early patient identification and market readiness.
The launch of Translarna for Duchenne patients 2 and older is planned to start as soon as the European Commission ratifies the CHMP recommendation.