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EMA hands UniQure orphan status first in Huntington's

The Dutch biotech expects to file the treatment for approval later this year

European Medicines Agency (EMA)

The European Medicines Agency (EMA) has granted UniQure’s investigational gene therapy an orphan medicinal product designation (OMPD).

Its Huntington’s disease candidate, currently known as AMT-130, becomes the first investigational AAV-gene therapy in this disease area to receive the status.

To qualify for OMPD in Europe, a therapy must be intended for the treatment of a disease that is life-threatening or chronically debilitating and have a patient prevalence in the European Union of no more than 5 in 10,000.

UniQure’s candidate fit the bill, not least because there are no currently approved medical treatments aimed at addressing the underlying cause of Huntington’s disease.

Matthew Kapusta, chief executive officer of UniQure, said: “The granting of orphan drug designation in Europe represents another important milestone for our AMT-130 programme.”

Back in October last year, the Food and Drug Administration (FDA) granted the candidate an orphan drug designation for the same indication.

Kapusta continued: “Huntington’s disease affects approximately 70,000 people in the US and Europe, making this one of the largest clinical unmet needs in the rare disease field.”

The Dutch biotech plans to file the drug - which consists of an AAV5 vector carrying out engineered micro-RNA designed to silence the Huntington gene - for review later this year.

The EMA’s move could help revive UniQure’s fortunes after it was last year forced to pull the EU’s first gene therapy due to a lack of demand.

Article by
Gemma Jones

25th January 2018

From: Regulatory



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