FDA Approves Novel Corticosteroid Treatment Option for DMD Patients

DMD Boy in wheelchair_iStock, ChiccoDodiFC

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

The FDA on Thursday approved Santhera Pharmaceuticals’ novel corticosteroid vamorolone, now to carry the brand name Agamree, for the treatment of Duchenne muscular dystrophy.

The announcement was made by Florida-based Catalyst Pharmaceuticals, which in June 2023 paid $90 million upfront and promised up to $141 million more in milestones to exclusively commercialize the drug in North America. As part of the deal, Santhera will now transfer the approved New Drug Application for Agamree to Catalyst, which expects to launch the product in the first quarter of 2024.

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

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

“The approval of Agamree underscores the potential of reshaping the DMD treatment paradigm for this life-threatening rare disease,” McEnany added.

DMD is a rare and genetic disorder characterized by progressive muscle weakness and degeneration, which typically affects the limbs and results in difficulties in walking and problems with balance. In its most progressed form, DMD can also affect the muscles of the heart and respiratory tract, often leading to their failure.

Patients with DMD are usually given glucocorticoid regimens, which are effective at slowing the progression of muscle deterioration, but also come with significant side effects.

While it is also a corticosteroid, Agamree works through a unique mode of action that elicits “differential effects” on glucocorticoid and mineralocorticoid receptors, modifying their downstream activities. This allows Agamree to maintain the typical efficacy 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 significantly superior time to stand from supine position velocity compared with placebo. The company supplemented this with data from three open-label studies including extension studies, showing a lower rate of adverse events versus current standard-of-care corticosteroids.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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