Wave Life Sciences Gets FDA Rare Pediatric Disease Designation for WVE-N531 to Treat Duchenne Muscular Dystrophy

Wave Life Sciences Ltd. (WVE), a clinical-stage biotechnology firm specializing in RNA-based medicines, recently announced that the U.S. Food and Drug Administration (FDA) has bestowed the Rare Pediatric Disease Designation upon its product WVE-N531. This designation targets the treatment of Duchenne muscular dystrophy (DMD) in boys who are suitable candidates for exon 53 skipping. WVE-N531 is being evaluated in the ongoing FORWARD-53 clinical trial, with significant data expected by the third quarter of 2024.

Anne-Marie Li-Kwai-Cheung, Chief Development Officer at Wave Life Sciences, emphasized the importance of this FDA designation, underlining the significant unmet needs in DMD and supporting Wave's innovative approach in developing treatments for rare diseases. According to Li-Kwai-Cheung, WVE-N531 aims to restore near full-length, functional dystrophin protein. Positive results from the FORWARD-53 trial could pave the way for additional programs targeting other exons, potentially benefiting up to 40% of boys with DMD.

WVE-N531 is an exon skipping oligonucleotide designed to stimulate the production of endogenous functional dystrophin protein. During the Part A study, which involved three doses of 10 mg/kg every other week, WVE-N531 exhibited an industry-leading mean exon skipping level of 53% and muscle tissue concentrations averaging approximately 42,000 ng/g. These concentrations are 20-30 times higher than those achieved by other exon-skipping technologies utilizing muscle delivery conjugates in DMD patients. Remarkably, the Part A data also showed distribution to myogenic stem cells, which are crucial for muscle regeneration. Notably, Wave Life Sciences is not aware of any other exon skipping or gene therapy that has demonstrated such uptake in myogenic stem cells. Preclinical studies in non-human primates further indicated that WVE-N531 achieved higher concentrations in the heart and diaphragm than in skeletal muscle, suggesting potential cardiac and respiratory benefits.

Currently, WVE-N531 is being evaluated in FORWARD-53, an open-label clinical trial involving 11 boys with DMD. The trial assesses dystrophin expression after 24 and 48 weeks of treatment, as well as pharmacokinetic, safety, and tolerability data.

The Rare Pediatric Disease Designation is awarded by the FDA for serious or life-threatening diseases that primarily affect individuals under 18 years of age and fewer than 200,000 people in the United States. If the New Drug Application for WVE-N531 is approved, Wave Life Sciences could receive a Priority Review Voucher.

Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by the absence or malfunction of dystrophin protein, essential for normal muscle function. Due to genetic mutations, individuals with DMD cannot produce functional dystrophin, leading to progressive muscle degeneration, including in the heart and lungs. Worldwide, DMD affects about one in 5,000 newborn boys, with 8%-10% of patients having mutations treatable with exon 53 skipping therapy. Exon skipping aims to address the root cause of DMD by promoting dystrophin production to stabilize or slow disease progression.

Wave Life Sciences is a biotechnology company focused on RNA medicines to transform human health. Its RNA platform, PRISM®, integrates multiple modalities, innovative chemistry, and genetic insights to create treatments for both rare and common disorders. Wave's diverse pipeline includes clinical programs for Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency, and Huntington’s disease, along with preclinical work in obesity. Headquartered in Cambridge, MA, Wave Life Sciences is driven by the mission to "Reimagine Possible," striving to eliminate the burden of disease.

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