FDA grants uniQure Regenerative Medicine Advance Therapy (RMAT) Designation

Today we are very happy in the Huntington community.  The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT)

“Designation for Investigational Gene Therapy AMT-130 in Huntington’s Disease is more than promising”, says President of IHA, Svein Olaf Olsen.  The RMAT ‘licence’ is based on three criteria.  The third criterion is what we notice in the Huntington’s family, namely that the results so far in the trial must be promising.  We know that the announcement and the FDA’s decision are based on 29 people who have participated in a clinical trial where the patient has been injected directly into the brain and in the area where Huntington’s ‘ravages’ at the beginning of the development of the disease. We’ve had many setbacks over the years. 

“Let’s hope that uniQure’s announcement is the beginning of good feedback from the pharmaceutical industry. In fact, we deserve it”, says Svein.

In making the announcement, Daniel Leonard, Senior Director of Global Patient Advocacy at uniQure said “ We look forward to continuing our partnership with the HD community to move the AMT-130 program forward as expeditiously as possible!”

Click here to read the full press release