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Wave Life Sciences granted FDA orphan drug designation for Huntington’s disease drug

pharmafile | June 22, 2016 | News story | Research and Development FDA, drug, huntington's disease, wave life sciences 

Wave Life Sciences (NASDAQ: WVE) has announced that it has received orphan drug designation for the US Food and Drug Administration (FDA) for WVE-120101, its lead candidate for the treatment of Huntington’s disease.

Huntington’s is a fatal, genetic, neurodegenerative disease that progressively breaks down nerve cells in the brain. It is estimated that 30,000 people in the US suffer from the disease while 200,000 carry the gene that causes the disease and are therefore at risk.

WVE-120101 targets a specific single nucleotide polymorphism (SNP) that is associated with the disease-causing mutation in the HTT gene. The drug enables selective silencing of the disease-causing HTT allele, while leaving its healthy elements free to produce normally functioning protein.

Orphan drug designation guarantees seven years of market exclusivity if approved, as well as providing other economic incentives for drug candidates in diseases affecting 200,000 or less people in the US.

Paul Bolno, CEO at Wave, says: “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 applications for both in late 2016.”

Sean Murray

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