Merck KGaA has received US FDA Fast Track designation for its generalised myasthenia gravis (gMG) drug treatment. Current treatments for gMG have limited efficacy and Merck’s cladribine capsules could become the first oral treatment for gMG, if approved.
gMG is a rare autoimmune neuromuscular disorder characterised by severe muscle weakness. It can affect any part of the body, but often presents in the voluntary muscles that control the eyes, eyelids, face and mouth. It can cause difficult moving, chewing, swallowing and occasionally even breathing.
Cladribine is designed to selectively target the B and T cells, which are believed to contribute to the production of harmful autoantibodies, causing inflammation and damage to the neuromuscular junction. The cladribine capsules are currently being studied in the global phase 3 MyClad trial. As part of the MyClad trial, Merck KGaA formed a patient council and works with more than 20 MG patient advocacy groups around the world.
“The FDA’s Fast Track designation, together with Orphan Drug designation, affirms that more treatments are needed for the gMG community,” said David Weinreich, global head of R&D and chief medical officer for Merck’s healthcare business. “Patient insights will continue to be instrumental to ensure we meet the needs of patients to deliver therapeutic innovation for this rare, chronic autoimmune neuromuscular disorder.”
The FDA also granted Orphan Drug designation to the cladribine capsules in June 2023, a special status granted to drugs which treat very rare diseases. The FDA’s Fast Track programme is designed to prioritise and accelerate the development of treatments for conditions with high unmet need. Merck will announce results from the MyClad trial following its completion.
In addition to the MyClad trial, Merck is also partnering with medical equipment manufacturing company Ad Scientiam to launch a prospective gMG-related study. The study will investigate the possible applications of wearable technologies, smartphone-based tests and electronic patient-reported outcomes in the management of gMG.