FDA Grants Fast Track Designation to Oral Treatment for Generalized Myasthenia Gravis

The Food and Drug Administration (FDA) has granted Fast Track Designation to cladribine capsules (EMD Serono, Boston, MA) for the treatment of individuals with generalized myasthenia gravis (gMG). Cladribine, which was previously granted Orphan Drug Designation by the FDA in June 2023, is designed to selectively target B and T lymphocytes involved in the autoimmune response driving gMG. According to a statement from EMD Serono, if approved, cladribine would be the first oral treatment for gMG.

Cladribine capsules are currently being evaluated in the global phase 3 MyClad clinical trial (NCT06463587), a randomized, double-blind, placebo-controlled study assessing the efficacy and safety of the investigational oral therapy in people with gMG (N=264). Participants will be randomized to receive either 2 brief treatment cycles of cladribine—administered at either a low or high dose—or a matching oral placebo. These treatment courses will be spaced 4 weeks apart. After the initial treatment phase, the trial will include a blinded extension and a retreatment period to evaluate the durability of response, determine the potential need for additional treatment, and monitor long-term safety. The primary outcome measure is change from baseline in the MG-Activities of Daily Living (MG-ADL) score at week 24 during the double-blind, placebo-controlled phase.

Source: EMD Serono Takes Patient-Directed Approach to Bring Innovation to the Treatment of Rare Neuromuscular Disorder, Generalized Myasthenia Gravis. Published 24 November, 2025. Accessed 3 December, 2025. https://www.businesswire.com/news/home/20251124788420/en/EMD-Serono-Takes-Patient-Directed-Approach-to-Bring-Innovation-to-the-Treatment-of-Rare-Neuromuscular-Disorder-Generalized-Myasthenia-Gravis.