Huntington’s Treatment Candidate Gets FDA Orphan Drug Designation

Huntington’s Treatment Candidate Gets FDA Orphan Drug Designation

Vaccinex recently announced that their drug candidate VX15 developed for the treatment of Huntington’s disease (HD) has received orphan drug designation from the U.S. Food and Drug Administration (FDA).

Orphan drug status is granted to therapies and biologics to treat rare diseases or disorders that impact fewer than 200,000 people in the U.S. or that are not expected to be profitable for pharmaceutical companies to develop and market. The status provides certain incentives in order to further drug development for the common good.

Vaxxinex’s drug candidate VX15 is an antibody that blocks the functional activity of a protein called semaphorin 4D (SEMA4D). Previous research on animal models has shown that SEMA4D triggers the activation of cells of the innate immune system within the central nervous system, called microglia and astrocytes. The chronic activation of these cells is thought to be implicated in the development of HD as well as other neurodegenerative conditions such as progressive multiple sclerosis (MS).

VX15 was tested previously in a Phase 1 clinical trial and was shown to be safe for people with MS. It is currently being tested in the Phase 2 clinical trial called SIGNAL to assess its safety and efficacy in people with HD.

“We are pleased to receive orphan drug designation from the FDA for VX15 as a potential treatment for Huntington’s disease,” Dr. Maurice Zauderer, chief executive officer of Vaccinex, said in a press release. “We hope that this study will contribute to treatment of HD, a serious disease for which there are, as yet, no approved disease-modifying therapies.”

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Scientists at the Martinos Center for Biomedical Imaging at Massachusetts General Hospital were able — for the first time — to track epigenetic factors, or non-genetic influences on gene activity, linked to Huntington’s disease in the brains of living humans.

The study, “Insights into neuroepigenetics through human histone deacetylase PET imaging,” published in the journal Science Translational Medicine, could advance the understanding of faulty brain processes in Huntington’s and in other neurodegenerative diseases and psychiatric diseases.

 

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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.

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