Potential DMD Therapy Now in Phase 1 Trial, WVE-210201, Likely To Be Named Orphan Drug in EU

The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), is recommending that WVE-210201 be designated an orphan drug as a potential Duchenne muscular dystrophy (DMD) treatment, its developer, Wave Life Sciences, announced.

WVE-210201 is an investigational compound of a type of therapy called exon skipping, which produces shorter but functional proteins. It is currently in a Phase 1 clinical trial (NCT03508947) now recruiting DMD patients at six sites in Europe and the U.S.

Exons are the bits of DNA that contain the information needed to generate proteins. Mutations in the DMD gene can disrupt how exons are read, which results in little or no functional dystrophin protein being produced. Dystrophin plays a crucial role in muscles’ function and protection, keeping muscle cells together and connected with surrounding structures.

Specifically, WVE-210201 is designed to “skip over” exon 51 in the DMD gene, an approach that could benefit about 13 percent of all DMD patients

Wave’s pre-clinical experiments have shown that WVE-210201 induces efficient exon 51 skipping and dystrophin restoration in DMD myoblasts — precursors of muscle cells — that were derived from DMD patients.

In the EU, treatment candidates are considered for orphan drug designation if:

• they aim to treat a life-threatening or chronically debilitating disease;
• the condition does not affect more than five in 10,000 people in the EU, or if the costs of developing the therapy are unlikely to be covered by the returns of its marketing;
• if the disease has no satisfactory method of diagnosis, prevention or treatment, or the investigative medication may provide a significant benefit or improvement.

The designation provides regulatory and financial incentives to develop and market treatments, including 10-year market exclusivity in the EU, reduced fees during development stages (like clinical testing), and direct access to centralized marketing authorization.

The European Commission is expected to soon adopt COMP’s recommendation.

“This positive opinion from the EMA’s [COMP], based on our in vivo data supporting medical plausibility and the potential for significant benefit of WVE-210201, marks another milestone in our efforts to try to improve the lives of boys suffering from [DMD],” Michael Panzara, MD, neurology franchise lead at Wave, said in a press release.

“We remain keenly focused on advancing WVE-210201 in our ongoing global Phase 1 clinical trial,” Panzara added.

The multicenter, double-blind, placebo-controlled Phase 1 study is assessing the safety, tolerability and plasma concentrations of intravenous WVE-210201 in ambulatory and non-ambulatory DMD patients (ages 5 to 18) who are amenable to exon 51 skipping.

Scientists will test single ascending doses of WVE-210201 in about 40 patients, and safety results are expected later this year. More information about this trial, including enrollment locations and contacts, is available here.