UniQure plans FDA filing for Huntington’s gene therapy after agency dispute
The company says FDA staff now view its Phase 1/2 data as enough to support an accelerated approval request for the experimental treatment.
By Daniel Okafor · Business Editor
3 min read
UniQure said it will ask the Food and Drug Administration to approve its experimental gene therapy for Huntington’s disease after a recent meeting with the regulator changed the path for its application. The update matters for patients with a rare inherited brain disease and for biotech investors watching how the FDA treats drugs for small patient groups.
The company said Wednesday that FDA officials indicated a three-year analysis from a Phase 1/2 study could support an accelerated approval request. UniQure now plans to file its application in the third quarter of this year, according to the company.
CNBC reported that UniQure shares jumped 70% on Wednesday after the announcement. The move followed months of public friction between the company and FDA leaders over whether the data behind the treatment were strong enough to justify a submission.
FDA stance shifts after earlier criticism
In March, UniQure said the FDA did not agree that data comparing patients who received the therapy with an outside control group were enough to support an application. CNBC reported that the agency’s position at the time made UniQure one of the more visible examples of drugmakers saying the FDA had changed earlier guidance, with rare disease companies affected in particular.
Then-FDA Commissioner Marty Makary discussed a treatment matching UniQure’s product in a February CNBC interview with Becky Quick, without naming the company. Makary said the agency had faced pressure to approve a treatment that showed “no benefit,” CNBC reported.
A senior FDA official later told reporters that the agency wanted UniQure to conduct a placebo-controlled trial to show the therapy “actually helps people,” according to CNBC. UniQure has said such a trial would be unethical because its treatment is delivered directly into the brain through surgery lasting several hours, and a placebo design could require patients to undergo a sham procedure.
Company relies on outside comparison
Rather than use a placebo group, UniQure compared patients treated with its gene therapy against expected Huntington’s disease progression using an external database. The company said that analysis showed the treatment slowed disease progression by 75% in the Phase 1/2 trial.
Huntington’s disease is a hereditary disorder that gradually damages nerve cells in the brain, CNBC reported. The condition is also known as Huntington’s chorea and is linked to a mutation in the HTT gene, according to the report.
UniQure said it now plans to use the same three-year analysis that had drawn scrutiny as the basis for its FDA application. Under accelerated approval, a drug can reach the market before final proof of clinical benefit if the company later confirms that benefit in another study.
The company said the FDA wants to agree on the design of that follow-up study before UniQure files its application. UniQure said the agency is considering a trial that would compare the treatment with the current standard of care rather than with a sham procedure, and the company expects to finalize those plans before submission.
The change comes after Makary left the FDA in May, CNBC reported. Other senior leaders also departed, including former Center for Biologics Evaluation and Research director Vinay Prasad and former Center for Drug Evaluation and Research director Tracy Beth Høeg.
CNBC reported that Replimune has also seen its regulatory prospects improve since those departures, with the company recently saying it would seek approval of its experimental melanoma drug for a third time.
This story draws on original reporting from CNBC.