WAVE Life Sciences Receives Orphan Drug Designation from FDA for its Lead Candidate Designed to Treat Huntington’s Disease

Allele-specific targeting silences mutant HTT allele while leaving healthy HTT allele intact On track to file investigational new drug (IND) application in late 2016

WAVE Life Sciences Ltd. (NASDAQ:WVE), a genetic medicines company focused on developing stereopure nucleic acid therapies for patients impacted by rare diseases, today announced that its lead candidate WVE-120101, which is being investigated for the treatment of Huntington’s disease (HD), has received Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). WVE-120101 targets rs362307, a Single Nucleotide Polymorphism (SNP) that is associated with the disease-causing mutation in the huntingtin (HTT) gene. WAVE’s approach enables selective silencing of the disease-causing HTT allele, while leaving the healthy HTT allele to produce normally functioning protein.

“We are pleased to receive Orphan Drug Designation for our first Huntington’s disease therapy and to advance what may be the first allele-targeted therapy into clinical trials, particularly as there are no approved disease-modifying treatments for HD,” said Paul Bolno, M.D., MBA, President and Chief Executive Officer of WAVE Life Sciences. “WVE-120101 is the first of our two lead allele-specific antisense programs for HD, and we are on track to file investigational new drug (IND) applications for both in late 2016. Each program distinctly targets the underlying genetic cause of HD and between these two programs, a substantial majority of HD patients would be covered. Our team at WAVE is driven by a sense of urgency and purpose as we work to advance disease-modifying treatments for HD patients.”

“Each potential new therapy for Huntington’s disease gives fresh hope to the more than 30,000 Americans symptomatic with HD and the 200,000 more who live fearfully at-risk of having inherited it,” said Louise Vetter, Chief Executive Officer of the Huntington’s Disease Society of America. “We are grateful for the commitment that WAVE Life Sciences has to improve the lives of families affected by this devastating neurological disease.”

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. Orphan drug designation would entitle seven years of market exclusivity in the United States if market approval is granted for WVE-120101. Additional incentives may include tax credits related to clinical trial expenses, an exemption from the FDA user fee, and FDA assistance in clinical trial design.


Source: WAVE Life Sciences 

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