A closely watched gene therapy for Huntington’s disease will get another chance at its first approval, giving its developer, UniQure, the chance to begin recovering from a major setback in the U.S.
UniQure said Thursday that it plans to submit a marketing application for the therapy, AMT-130, in the U.K. later this year following a positive meeting with drug regulators there. That submission will be based on a three-year analysis from ongoing trials in the U.S. and Europe that have shown an apparent slowing of disease progression at the highest dose tested.
“This is an important milestone for the Huntington’s disease community and we remain committed to working closely with regulators globally, with the goal of bringing this potentially transformative therapy to patients in the U.K. and internationally,” said UniQure CEO Matt Kapusta, in a statement.
The Netherlands-based gene therapy maker also revealed that an important meeting with the Food and Drug Administration will take place in the second quarter. The agency recently demanded that UniQure run a new trial before considering approval of AMT-130, leaving its path forward in the U.S. unclear. UniQure and the FDA will discuss “key elements” of a potential Phase 3 trial design at that meeting, and the agency will offer feedback on a four-year study analysis coming in the third quarter. The company is eyeing possible approvals in other countries, too, and will provide an update later this year.
The news represents newfound hope for one of the most advanced treatments in testing for Huntington’s, a progressive and debilitating neurodegenerative condition without a cure. Available treatments available only manage disease symptoms.
UniQure’s AMT-130 silences a gene that produces a mutated protein linked to nerve damage. Over the years, the company has shared a series of data updates demonstrating the therapy’s potential to slow disease progression. Results disclosed last year stunned researchers and sent company shares soaring. UniQure said it intended to seek an accelerated U.S. approval afterwards.
But the FDA wasn’t receptive to UniQure’s request, which the company claimed to be a shift from prior feedback. The agency then demanded UniQure run a new "sham-controlled" trial, a situation analysts referred to as a “worst-case scenario” for the company. UniQure and the FDA then engaged in a public spat, one of multiple recent conflicts between the agency and the developers of a rare disease medicine.
Progress towards a filing in the U.K., then, represents an “important symbolic win,” wrote Leerink Partners analyst Joseph Schwartz. While the U.K. isn’t necessarily known for “strong reimbursement,” the development could be “the beginning of positive news” for the company.
Stifel analyst Paul Matteis noted that the situation in the U.K. could “have some impact” on the therapy’s prospects in the U.S., particularly given the political pressure the FDA is under to be more flexible with rare disease treatments.
UniQure shares climbed by more than 20% Thursday morning.
