FDA Approves Generic Emflaza for Duchenne Muscular Dystrophy

On Friday, June 14, 2024, the U.S. Food and Drug Administration (FDA) announced the approval of the first generic version of Emflaza (deflazacort) oral suspension designed for the treatment of Duchenne muscular dystrophy (DMD). Duchenne muscular dystrophy is a severe and rare genetic disorder characterized by progressive muscle degeneration and weakness, predominantly affecting young boys.

Deflazacort oral suspension is classified as a corticosteroid and is prescribed for patients aged 5 and older. However, it is not suitable for individuals with a known hypersensitivity to deflazacort. The most frequently reported side effects among patients include a Cushingoid appearance, weight gain, increased appetite, upper respiratory tract infections, coughing, frequent urination, excessive hair growth, central obesity, and nasopharyngitis.

Ketan Mehta, the founder and CEO of Tris Pharma, expressed the significance of this FDA approval in a public statement. He highlighted the critical need for more accessible treatment options for DMD, given the limited alternatives currently available. Mehta emphasized that the introduction of a generic therapy could profoundly impact patients, caregivers, and healthcare providers who rely on this medication for managing the disease.

The FDA granted the approval for the generic version of Emflaza oral suspension to Cranbury Pharmaceuticals, which is a subsidiary of Tris Pharma. This development marks a noteworthy milestone in addressing the treatment needs for those affected by Duchenne muscular dystrophy.