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uniQure Receives FDA RMAT Designation for Investigational Gene Therapy AMT-130 in HD

Uniqure‘s gene therapy for Huntington’s Disease, named AMT-130 has passed a significant next step.

Today the company announced that the US regulators, FDA, has granted them a Regenerative Medicine Advanced Therapy (RMAT) designation. 

The designation is given based on 3 criteria:

a) The treatment is categorized as regenerative;

b) The treatment is supposed to treat a life-threatening disease;

c) The preliminary evidence indicates that the drug has the potential to address unmet medical needs for the disease.

“This is great news and another step in the right direction for the HD community”, states Astri Arnesen, President of EHA.  

The FDA designation is based on positive evaluation of the preliminary study results.  The results have not yet been shared with the public, but will be soon, according to Uniqure’s press release today.  

“I am thrilled by this announcement and cross my fingers for a fast and efficient continuation of the study in order to get sufficient proof of the potential efficacy of the treatment. Only around half of the applications for a RMAT are approved, and the fact that AMT-130 was evaluated positively by the regulators nurture my hope for the future”, says Arnesen with a big smile.