FDA Approves New Corticosteroid Treatment Option for DMD Patients | BioSpace

Pictured: A boy sitting in a wheelchair on a beach/iStock, ChiccoDodiFC

The FDA on Thursday approved Santhera Pharmaceuticals’ new corticosteroid vamorolone, now marketed under the brand name Agamree, for the treatment of Duchenne muscular dystrophy.

The announcement was made by Florida-based Catalyst Pharmaceuticals, which paid $90 million upfront in June 2023 and pledged up to $141 million more as milestones to exclusively market the drug in America North. As part of the agreement, Santhera will now transfer the approved new drug application for Agamree to Catalyst, which plans to launch the product in the first quarter of 2024.

Agamree is an oral suspension medication indicated for patients with Duchenne muscular dystrophy (DMD) aged two years and older. Its label does not have a boxed warning, but does include precautions regarding alterations in endocrine, cardiovascular and renal function, immunosuppression, ophthalmic side effects, increased risk of infections, as well as mood disorders and behavior.

In a statement, Catalyst CEO Patrick McEnany said Agamree has transformative potential and could potentially make a significant difference for patients living with DMD and potentially other chronic inflammatory diseases.

The approval of Agamree highlights the potential to reshape the DMD treatment paradigm for this rare, life-threatening disease, McEnany added.

DMD is a rare genetic disease characterized by progressive muscle weakness and degeneration, which usually affects the limbs and leads to difficulty walking and balance problems. In its most advanced form, DMD can also affect the muscles of the heart and airways, often leading to their failure.

Patients with DMD typically receive glucocorticoid regimens, which are effective in slowing the progression of muscle deterioration but also carry significant side effects.

Although also a corticosteroid, Agamree acts through a unique mode of action that causes differential effects on glucocorticoid and mineralocorticoid receptors, thereby altering their downstream activities. This allows Agamree to maintain the typical effectiveness of glucocorticoid regimens while also achieving a better tolerated side effect profile.

Santhera supported its FDA application with data from the pivotal Phase IIb VISION-DMD study. Patients treated with Agamree showed a significantly greater speed of rising from a supine position compared to placebo. The company supplemented this data with data from three open-label studies, including extension studies, showing a lower rate of adverse events than current standard-of-care corticosteroids.

Tristan Manalac is a freelance science writer based in Metro Manila, Philippines. He can be contacted at tristan@tristanmanalac.com or tristan.manalac@biospace.com.