FDA Releases Guidance on Drug Development for Fabry Disease

The FDA has okayed the use of a single trial to prove efficacy of any drug developed to treat Fabry Disease, according to a draft guidance from the agency.

Sponsors should think about combining multiple clinical outcomes in their assessments given FD’s multi-systemic nature, highly variable rate of progression and heterogeneity, advises the guidance which focuses on trial design, efficacy endpoints and eligibility criteria.

If the drug is meant to slow or arrest disease progression but not reverse it, the trial should last long enough to observe disease progression in the control group, the agency says.