By Michael Erman and Puyaan Singh
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 the gene therapy.
On Monday, the Dutch drugmaker said the U.S. Food and Drug Administration had called for a new study to support the approval of its gene therapy for the rare, inherited neurodegenerative disorder and rejected its most recent trial for not including participants given a placebo.
The FDA official, who spoke on the condition of anonymity, said a trial run by the company several years ago with a placebo design “was stone cold negative. We have a failed product here.”
The call followed criticism from UniQure and patient advocate groups of the FDA’s request.
The official also accused UniQure of making a “distorted or manipulated comparison” in how it presented data for the treatment.
UniQure declined to comment on the FDA official’s remarks.
The company’s U.S.-listed shares rose on Thursday ahead of the media briefing by more than 20% after recent losses. They lost most of those gains and were up 8% at $9.80 in afternoon trading.
UNACCEPTABLE COMPARISON
UniQure in September said its studies showed statistically significant improvement in slowing disease progression after three years by 75%, based on a clinical rating scale when compared with patients from an external dataset of Huntington’s patients.
The FDA official said such a comparison was not acceptable and that the agency has “never in 25 years” accepted such a trial design for Huntington’s disease therapies.
“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.
(Reporting by Michael Erman, Puyaan Singh, Christy Santhosh, Mrinalika Roy in Bengaluru; Editing by Krishna Chandra Eluri and Bill Berkrot)
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