Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy
Lead SponsorMarathon Pharmaceuticals
Study TypeExpanded Access
StatusApproved for marketing
Indication/ConditionDuchenne Muscular Dystrophy
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.
Deflazacort will be supplied free of charge and shipped directly to patients or caregivers, as appropriate. Treating physicians will continue to follow their standard of care activities and procedures for management of DMD. Treating physicians participating in the expanded access program are required to collect/document any patient or caregiver reported safety events and report to the sponsor..
Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized immediately to the active metabolite, 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone.
Main Inclusion Criteria: Confirmed diagnosis of Duchenne muscular dystrophy The patient is ≥ 5 years old Current on all childhood vaccinations including the chicken pox vaccine Main Exclusion Criteria: History or current medication condition that could affect safety or poses an additional risk Hypersensitivity or allergic reaction to steroids or their formulations