Jan Klatt, MD, Head of Development Unit Neurology & Immunology, Merck Healthcare, Germany, discusses the U.S. Food and Drug Administration’s (FDA) Fast Track designation of cladribine capsules for patients with generalized myasthenia gravis (gMG).
gMG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine. The exact reason this occurs is not known.
The U.S. FDA has granted Fast Track designation for cladribine capsules for the treatment of gMG. Fast Track designation is designed to facilitate the development and accelerate the review of therapies for conditions with severe unmet needs. The therapy was previously granted Orphan Drug Designation in 2023.
Cladribine is currently being studied in the global phase 3 MyClad clinical trial (NCT06463587), a global Phase 3, randomized, double-blind and placebo-controlled study designed to assess the safety and efficacy of cladribine capsules in 264 patients with gMG. Cladribine is designed to selectively target B and T cells believed to contribute to the production of harmful autoantibodies that cause inflammation and neuromuscular junction damage.
Results of this trial are expected to be announced upon completion. If approved, cladribine capsules could be the first oral treatment for gMG.
In addition to this designation, collaborations with patient organizations and Ad Scientiam, a medical equipment manufacturing company, to support patient-directed approaches to gMG treatment are ongoing.
To design the MyClad trial and ensure it meets the needs of patients, a patient council was developed and collaborations with over 20 patient advocacy groups around the world were initiated. Insights from the council have informed study protocols, recruitment materials, and potential future product packaging options for the clinical trial program.
The collaboration with Ad Scientiam is also working to launch a prospective, multi-center pilot study exploring an investigational software as a medical device designed to support the management of gMG. The study will investigate how wearable technologies, smartphone-based functional tests, and electronic patient-reported outcomes (ePROs) could detect changes in disease trajectory in real-world conditions.
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To learn more about gMG and other rare neurology conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/
