Merck KGaA, a global leader in science and technology, has announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for its investigational cladribine capsules to treat generalized Myasthenia Gravis (gMG), a rare and debilitating autoimmune neuromuscular disorder.
The move builds on the FDA’s Orphan Drug Designation for cladribine, granted in June 2023, highlighting the urgent need for new therapies for this chronic condition. If approved, cladribine could become the first oral treatment for gMG, a disease that causes severe muscle weakness and drastically affects patients’ daily lives.
“The FDA’s Fast Track designation, alongside Orphan Drug status, underscores the pressing need for new gMG treatments,” said David Weinreich, MD, Global Head of R&D and Chief Medical Officer, Healthcare, Merck KGaA. “We are committed to putting patients at the center of our program, ensuring their voices shape the future of therapeutic innovation for this rare disorder.”
Merck is actively engaging with the gMG community, forming a patient council and collaborating with over 20 global advocacy groups. Patient insights have guided study protocols, recruitment materials, and potential product packaging, ensuring the upcoming MyClad Phase 3 trial addresses real-world patient needs.
In parallel, Merck is working with medical technology company Ad Scientiam to pilot a study using wearable devices, smartphone tests, and electronic patient-reported outcomes (ePROs) to track disease progression in real-world settings.
The MyClad trial is a global, randomized, double-blind, placebo-controlled Phase 3 study evaluating cladribine’s safety and efficacy in 264 gMG patients. Cladribine is designed to selectively target B and T cells, reducing the harmful autoantibodies that damage neuromuscular function. Its short-course, home-based oral regimen could significantly ease the treatment burden for patients.
Merck KGaA will share trial results once available and remains committed to ongoing collaboration with the gMG community to ensure patient needs guide future clinical developments.
