FDA Grants MDA Kickstart Program Rare Pediatric Disease Designation and Orphan Drug Designation in Congenital Myasthenic Syndrome

Published Date: 22 Dec 2024

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

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Real-world 12‑month results from WeSMA, a phase 4 study, showed that oral risdiplam is well tolerated in adults with SMA, with stable motor function and consistent safety findings.

A phase 4 study is evaluating whether adults with chronic inflammatory demyelinating polyneuropathy can transition from intravenous immunoglobulin to subcutaneous efgartigimod PH20 without first experiencing disease worsening.

A new phase 4 trial, OCTAGON, will test a rapid, predefined corticosteroid-tapering schedule in patients with generalized myasthenia gravis treated with ravulizumab.

New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

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