Wave Life Sciences Announces Fast Track Designation from U.S. FDA for Suvodirsen
Suvodirsen is an investigational stereopure oligonucleotide in development for the treatment of DMD patients amenable to exon 51 skipping
Clinical dystrophin data from interim analysis of ongoing OLE study on track for 4Q 2019
DYSTANCE 51 global Phase 2/3 trial is underway
“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
Fast Track designation is granted by the
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 U.S. 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
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.
About Duchenne Muscular Dystrophy
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.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the anticipated benefits of having received Fast Track designation for suvodirsen, the expected timing and plans to report interim data from the ongoing OLE, the intention to use the results from the OLE and Phase 2/3 trials to seek various regulatory approvals for suvodirsen globally, and the anticipated timing of such regulatory filing in
Source: Wave Life Sciences