FDA grants rare pediatric disease designation for Duchenne muscular dystrophy treatment

The FDA has granted rare pediatric disease designation to an investigational oligonucleotide for the treatment of boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping, according to the manufacturer.

In a press release, Wave Life Sciences said the exon-skipping therapeutic, WVE-N531, is currently being evaluated in the FORWARD-53 clinical trial, a registrational, open-label study that includes 11 boys with the condition.

Primary study endpoints for the phase 2 study investigating the novel therapeutic — which is designed to induce production of endogenous, functional dystrophin protein — include dystrophin expression after 24 and 48 weeks of 10 mg/kg doses, as well as safety, tolerability and pharmacokinetic data.

The FDA confers rare pediatric disease designation to potential therapies that address either serious or life-threatening diseases in those aged 18 years or younger and affect fewer than 200,000 people in the United States.

Should a new drug application for WVE-N531 be approved by the FDA, Wave would be eligible to receive a priority review voucher, per the release.

“This designation from FDA underscores that significant unmet needs remain,” Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, chief development officer at Wave Life Sciences, said in the release. “With our WVE-N531 program, we are aiming to restore clinically meaningful levels of near full length, functional dystrophin protein.”

According to the release, Wave expects to deliver data, including 24-week dystrophin protein expression from muscle biopsies, during the third quarter of 2024.