Wave Life Sciences Announces Fast Track Designation from US FDA

Wave Life Sciences Ltd. recently announced the US FDA has granted Fast Track designation to suvodirsen for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. The designation was based on comprehensive in vitro and in vivo nonclinical data that support the potential for suvodirsen to address a significant unmet medical need.

“Our goal for Wave’s Duchenne programs is to urgently develop therapies that restore functional dystrophin to levels that have the potential to result in meaningful clinical benefit,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We are thrilled to have received Fast Track designation from the FDA for suvodirsen, a decision which we believe reflects its potential to provide a meaningful option for those living with this dreadful genetic disease. We look forward to continuing to work with regulatory authorities with the hope of bringing suvodirsen to the Duchenne community as quickly as possible.”

Fast Track designation is granted by the FDA for products that are intended for the treatment of serious or life-threatening disease or conditions, which demonstrate the potential to address an unmet medical need. The designation offers the opportunity for frequent interactions with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, as well as eligibility for rolling submission of a New Drug Application (NDA).

Suvodirsen is currently being evaluated in an ongoing open-label extension (OLE) study for DMD patients amenable to exon 51 skipping. Wave remains on track to deliver an interim analysis of dystrophin expression from muscle biopsies in boys receiving suvodirsen in the OLE study in the fourth quarter of 2019. Pending positive clinical dystrophin expression data, the company expects to file for an accelerated approval of suvodirsen in the US in the second half of 2020. Suvodirsen is also currently being studied in DYSTANCE 51, a global Phase 2/3, multicenter, randomized, double-blind, placebo-controlled trial that will evaluate the efficacy and safety of suvodirsen. Results from the DYSTANCE 51 trial are intended to support global regulatory filings for suvodirsen. The study is also the first ever selected by the FDA for its Complex Innovative Trial Design (CID) pilot program.

Suvodirsen has also been granted orphan drug designation for the treatment of DMD by the FDA and the European Commission, as well as rare pediatric disease designation by the FDA. In addition to suvodirsen, Wave continues to advance WVE-N531, its preclinical candidate to treat DMD in boys amenable to exon 53 skipping. The company is also exploring exon targets beyond those targeted by suvodirsen and WVE-N531, including exons 44, 45, 52, 54, and 55.

Suvodirsen is an investigational stereopure oligonucleotide currently being evaluated in an ongoing open-label extension (OLE) study for the treatment of boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Suvodirsen is also being a studied in DYSTANCE 51, a global Phase 2/3, multicenter, randomized, double-blind, placebo-controlled trial that will evaluate the efficacy and safety of suvodirsen.

Approximately 13% of DMD patients have genetic mutations that are amenable to treatment with an exon 51 skipping therapy. Exon-skipping technology has the potential to induce cellular machinery to ‘skip over’ a targeted exon and restore the reading frame, resulting in the production of internally truncated, but functional dystrophin protein.

Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular disorder caused predominantly by out-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs. Worldwide, DMD affects approximately one in 5,000 newborn boys.

Wave Life Sciences (NASDAQ: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. For more information, visit www.wavelifesciences.com.