DARMSTADT, GERMANY -- Merck, a leading science and technology company, announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for cladribine capsules for the treatment of the rare, chronic autoimmune neuromuscular disorder, generalized Myasthenia Gravis (gMG). In June 2023, the FDA granted Orphan Drug Designation for cladribine capsules for the treatment of gMG. In addition, the Company is actively collaborating with patient organizations and Ad Scientiam, a medical technology company, to support a patient-directed approach to the future management of gMG.

If approved, cladribine capsules, currently being studied in the global Phase 3 MyClad trial, could become the first oral treatment for gMG, a rare autoimmune neuromuscular disorder that causes severe muscle weakness and significantly impacts patients’ lives. The FDA’s Fast Track program is designed to facilitate the development and accelerate the review of therapies for serious conditions with unmet medical needs, potentially bringing treatments to patients sooner. The FDA Orphan Drug Designation is designed to encourage the development of treatments for rare diseases or conditions, often defined as those affecting fewer than 200,000 patients in the U.S.

“The FDA’s Fast Track designation, together with Orphan Drug Designation, affirms that more treatments are needed for the gMG community,” said David Weinreich, MD, Global Head of R&D and Chief Medical Officer, Healthcare business of Merck. “As a patient-directed company, we are prioritizing the voices of MG patients in shaping our clinical program. Their 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.”

To shape the MyClad trial and ensure it meets the needs of patients, Merck developed a patient council and collaborates with more than 20 MG patient advocacy groups around the world. Insights from the council have informed study protocols, recruitment materials and potential future product packaging options for the clinical trial program.

In addition, Merck is collaborating with Ad Scientiam, a medical equipment manufacturing company, to launch a prospective, multi-center pilot study exploring an investigational software as a medical device designed to support the management of people living with 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.

The MyClad trial (NCT06463587) is a global Phase 3, randomized, double-blind and placebo-controlled study designed to assess the efficacy and safety of cladribine capsules in 264 patients with gMG. Cladribine is proposed to target selectively the B and T cells believed to contribute to the production of harmful autoantibodies that cause inflammation and damage to the neuromuscular junction. This mechanism of action, combined with a short-course infrequently-administered oral regimen taken at home, that if approved, may help reduce the burden of the disease by addressing its underlying cause.

Merck will announce results from the MyClad trial following its completion and remains committed to ongoing collaboration with the gMG community to ensure patient needs continue to guide clinical developments.