uniQure set for May FDA verdict on Huntington's gene therapy
uniQure chief executive Matt Kapusta.
The FDA is due to deliver a decision on uniQure's gene therapy for neurodegenerative disorder Huntington's by 1st May next year, after granting a priority review for the candidate.
The FDA's acceptance of the marketing application marks a turnaround for the ifezuntirgene inilparvovec (AMT-130) programme, which the agency knocked back with a refusal-to-file notification last year.
That said, uniQure has revealed that it will have to face an advisory committee meeting to review the data for the gene therapy, which may explain why shares in the biotech slipped around 7.5% after the announcement.
In September, the stock sank after four-year follow-up data from the phase 1/2 trial submitted in support of the filing seemed to suggest its efficacy may have been starting to tail off. That may be taken into account at the advisory committee meeting, which so far has no date allocated, although the review will focus on an earlier three-year readout.
In a statement, uniQure said the start of the priority review is an "important milestone" for patients with Huntington's disease, as AMT-130 could become the first disease-modifying treatment for the devastating disorder.
"With the FDA's priority review underway, we will work in conjunction with the agency, clinicians, and the Huntington's community to support the review process," said chief executive Matt Kapusta, who added that a confirmatory trial – which would be a condition of accelerated approval – is due to start screening patients before the end of the year.
Updated three-year data from the phase 1/2 trial reported in September, in 15 subjects, showed improvements of 80% on the cUHDRS scale and 67% using the TFC scale, which both measure disability progression.
AMT-130 has breakthrough therapy and regenerative medicine advanced therapy (RMAT) designations from the FDA, as well as fast-track status. Meanwhile, uniQure has also submitted the gene therapy for approval in the UK.
There is intense interest in the progress with AMT-130, as the current approved treatments for the neurodegenerative disorder – Ingrezza (valbenazine), Austedo (deutetrabenazine), and Xenazine (tetrabenazine) – are all VMAT2 inhibitors designed to treat the movement disorders (chorea) that characterise the disease, rather than the underlying pathology.
Other phase 3 candidates for Huntington's include Novartis' small-molecule huntingtin pre-mRNA splicing modifier votoplam in the INVEST-HD study, and Prilenia Therapeutics' oral sigma-1 receptor agonist pridopidine in the PRECISE-HD study. Both are designed to reduce levels of neurotoxic forms of the huntingtin protein that accumulate in the disorder.
