In another sign that the FDA is easing its stance toward rare disease drug candidates under new leadership, the agency will reconsider Regenxbio’s gene therapy for an ultra-rare childhood disease, a reversal coming just about four months after a rejection.
The Maryland biotech announced Monday that the FDA has agreed that existing clinical data are sufficient to support an accelerated approval filing for Navsunli (clemidsogene lanparvovec), a one-time gene therapy for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
The agency has reversed its previous demand that Regenxbio enroll more patients and incorporate an untreated control arm rather than relying on natural history data, requirements that the company had flagged as difficult for an ultra-rare disease population.
Now, backtracking on its demands, the FDA has asked for a meeting with Regenxbio to review existing longer-term biomarker and clinical data, and will review a resubmission “on an expedited basis,” according to the company.
In the letter, the agency asked that any future resubmission should include updated safety summary and a benefit-risk assessment, including vector integration analysis in MPS programs.
Sources:
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