March 5 (Reuters) – A senior FDA official called uniQure’s experimental treatment for Huntington’s disease a “failed product” in a conference call with media members on Thursday, casting further doubt on the prospects of its experimental gene therapy.
“We have a randomized trial within one year which was stone cold and negative. We have a failed product here,” the official said on the call, which was held in response to criticism from the company and patient advocate groups.
The official also accused uniQure of making a “distorted or manipulated comparison” in how it presented data for the treatment.
UniQure’s studies last year showed a statistically significant improvement in slowing the progression of the disease after three years based on a clinical rating scale when compared with patients from an external dataset of Huntington’s patients. The reported data suggested AMT-130 slowed disease progression by about 75%.
However, the FDA official said such a comparison was not acceptable.
“We do not dispute the claim that it is 75% better than those people, what FDA disputes is that those people are a fair comparator,” the official said.
UniQure did not immediately respond to a Reuters request for comment.
(Reporting by Michael Erman, Puyaan Singh, Christy Santhosh, Mrinalika Roy in Bengaluru; Editing by Krishna Chandra Eluri)
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