EU panel recommends expanded use of DMD treatment Agamree

The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the European Union be expanded to include people with Duchenne muscular dystrophy (DMD) as young as 2.

The CHMP is an arm of the European Medicines Agency (EMA) tasked with reviewing data on experimental therapies. The committee’s recommendation will be reviewed by the European Commission, which has final say over therapy approvals in the EU. The commission isn’t required to follow the CHMP’s recommendations, but it almost always does.

“This positive CHMP opinion marks an important step towards our goal of making Agamree available to every DMD patient who could benefit,” Dario Eklund, CEO of Agamree’s developer, Santhera Pharmaceuticals, said in a company press release.

DMD is a genetic disorder marked by progressive muscle damage. For decades, a mainstay part of DMD treatment has involved corticosteroids, anti-inflammatory medications that work by mimicking the stress hormone cortisol. Corticosteroid treatment has been shown to help slow DMD progression, allowing patients to retain more motor function for longer. However, long-term use of corticosteroids often causes unpleasant side effects such as weight gain, skin problems, and mood changes. Corticosteroid treatment in children also often leads to stunted growth.

“For families and clinicians, deciding when to initiate treatment in very young children can be particularly challenging, given the limited treatment options available,” said Eklund.

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Agamree is a dissociative corticosteroid, meaning it binds to the same receptors as standard corticosteroids, but in a way expected to retain their anti-inflammatory effects while minimizing growth stunting and other side effects.

Agamree was approved in the EU in late 2023 to treat people with DMD ages 4 and older. The therapy has also been approved for that indication in the U.K., China, and Canada. In the U.S., Agamree has been approved since 2023 to treat DMD patients as young as 2. The therapy is marketed in North America by Catalyst Pharmaceuticals.

Treatment approvals were based mainly on data from the VISION-DMD clinical trial (NCT03439670) , which tested Agamree against traditional corticosteroids or a placebo in boys with DMD ages 4 to 6. Results indicated that Amagree is comparable to traditional steroids in its ability to preserve muscle function, and patients who switched from traditional corticosteroids to Agamree saw no change in efficacy. Safety findings were also positive, including the maintenance of a normal growth trajectory.

Santhera did not say which data underpinned the CHMP’s recommendation to expand approval. The company noted that starting treatment very early can offer the greatest benefit, but the use of traditional corticosteroids in toddlers raises concerns.

“Initiating anti-inflammatory therapy early in DMD has the potential to reshape the long-term trajectory of the disease,” said Shabir Hasham, MD, chief medical officer of Santhera. “The CHMP’s positive opinion reflects the strength of the AGAMREE clinical and safety data and recognizes the need for better tolerated treatment options in young children. We remain committed to providing clinicians and families with a treatment option for this vulnerable population.”