WAVE Life Sciences Gets Orphan Drug States From FDA For Lead Candidate To Treat Huntington's Disease

WAVE Life Sciences Ltd. WVE revealed that its lead candidate WVE-120101, which is being investigated for the treatment of Huntington's disease (HD), has been accorded with Orphan Drug Designation by the Food and Drug Administration (FDA). According to the company, WVE-120101 targets rs362307, a Single Nucleotide Polymorphism (SNP) that is associated with the disease-causing mutation in the huntingtin (HTT) gene.

WAVE Life Sciences' approach allows selective silencing of the disease-causing HTT allele while leaving the healthy HTT allele to make normally functioning protein. The company pointed out that the Orphan Drug Act offered 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.

The company's President and CEO, Paul Bolno, said, "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. 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."

Huntington's Disease Society of America CEO, Louise Vetter, reacted to the development saying, every potential new therapy for Huntington's disease offered 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.