uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today in a press release on their official website announced that AMT-130, its proprietary gene therapy candidate for Huntington's disease, has received an Orphan Medicinal Product Designation from the European Medicines Agency. AMT-130 is the first investigational AAV-gene therapy in Huntington's disease to receive such designation. In October 2017 the company announced that the U.S. Food and Drug Administration had also granted orphan drug designation for the same therapy. There are currently no approved medical treatments aimed at addressing the underlying cause of Huntington's disease, and AMT-130 has the potential to play a role in this area of high unmet medical need.
AMT-130 consists of an AAV5 vector carrying an engineered micro-RNA specifically designed to silence the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. Using AAV vectors to deliver micro-RNAs directly into the brain represents a highly innovative approach to treating Huntington's disease.
"The granting of orphan drug designation in Europe represents another important milestone for our AMT-130 program," said Matthew Kapusta, chief executive officer of uniQure. "Huntington's disease affects approximately 70,000 people in the U.S. and Europe, making this one of the largest clinical unmet needs in the rare disease field. We expect to file an Investigational New Drug application later this year and be the first AAV-gene therapy to enter clinical development for Huntington's disease."
To qualify for OMPD (Orphan Medicinal Product Designation) 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. OMPD offers product market exclusivity for ten years in the European Union following regulatory approval, along with tax and financial incentives for companies developing medicines for such orphan indications.
Additional information - uniQure
Watch uniQure's YouTube video below.