Wave Life Sciences Receives US Orphan Drug and Rare Pediatric Disease Designations for WVE-210201

Wave Life Sciences (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD). The European Commission previously granted orphan drug designation for WVE-210201 in July 2018.

As quoted in the press release:

“Our team is motivated by a sense of urgency and compassion for the patients, families and caregivers affected by Duchenne muscular dystrophy and other serious, life-threatening conditions with high areas of unmet need,” said Michael Panzara, MD, MPH, Neurology Franchise Lead of Wave Life Sciences. “We are very pleased to receive these two important designations from the FDA and believe they further reinforce the potential of WVE-210201 to help boys suffering from DMD.”

The Orphan Drug Act provides for economic incentives to encourage the development of drugs intended to treat, diagnose or prevent rare diseases and conditions affecting fewer than 200,000 people in the United States. In determining orphan drug designation, the FDA’s Office of Orphan Products Development evaluates preclinical and clinical data to identify products as promising for rare disease. If market approval is granted by the FDA for WVE-210201 for the treatment of DMD, orphan drug designation would entitle Wave to seven years of market exclusivity in the United States. Additional incentives may include tax credits related to clinical trial expenses, exemption from prescription drug user fees and FDA assistance in clinical trial design.

Click here to read the full press release.