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FDA grants orphan drug status to UniQure’s Huntington’s Disease treatment

Becomes first investigational gene therapy to receive ODD for the disease


Dutch biotech UniQure has won orphan drug designation (ODD) for its investigational treatment for Huntington’s Disease, a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioural abnormalities and cognitive decline.

The drug - currently known as AMT-130 - could potentially be the first treatment approved for the disease in the US, with UniQure looking to bring the therapy to clinical trials next year.

This would be a big win for the biotech, as the US Food and Drug Administration (FDA) could offer US market exclusivity for up to seven years thanks to the ODD programme - which provides a ‘special’ status for investigational drugs that are developed for rare disease.

Matthew Kapusta, chief executive officer, UniQure, said: “Attaining orphan designation recognises the potential that AMT-130 holds in delivering meaningful therapeutic benefit to patients suffering from this devastating disease.”

The group published results from preclinical studies last year suggesting that a one-time administration of AAV5-delivered therapy into the central nervous system could block the mutant HTT gene that causes the disease.

According to UniQure, AMT-130 does exactly that as the therapy consists of an AAV5 vector that carries an artificial micro-RNA, which ‘silences’ the Huntington gene when it reaches the brain.

The designation supports UniQure’s “ongoing development in Huntington’s”, which is a therapy area the group has long been interested in, alongside haemophilia and cardiovascular diseases.

Article by
Gemma Jones

9th October 2017

From: Regulatory



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