Vamorolone recommended for approval in EU as DMD treatment

Final decision in Europe on newer steroid type expected by year's end

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A European Medicines Agency (EMA) committee has recommended that vamorolone be approved in the European Union to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older.

The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), now will be reviewed by the European Commission, which is expected to make a final decision on vamorolone before the year’s end. While the EU regulatory body isn’t require to abide by the CHMP’s recommendations, it usually does.

The commission’s final decision would apply to all EU member states, as well as Iceland, Liechtenstein and Norway. Assuming that vamorolone is approved, Santhera Pharmaceuticals, which has marketing rights, plans to first commercially launch the therapy in Germany in early 2024. Santhera plans to sell vamorolone under the brand name Agamree.

“We are thrilled about the CHMP’s positive opinion, which recognizes the urgent medical need for an effective and well tolerated treatment for this devastating disease,” Dario Eklund, CEO of Santhera, said in a company press release.

“We can now execute on our plans to ensure AGAMREE is made available to patients in the EU as soon as the European Commission marketing authorization is received,” Eklund said.

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The positive recommendation by the CHMP also was welcomed by the greater muscular dystrophy community in Europe, and by researchers.

“We are very excited about the positive opinion from the European Medicines Agency” committee,” said Ezio Magnano, president of the support organization Parent Project Italy.

“[This] announcement is another huge step ahead for our Duchenne and Becker community,” Mangnano said.

Maggie Walter, MD, an associate professor at Ludwig-Maximilians-University of Munich, in Germany, said vamorolone “offers Duchenne patients a new treatment alternative to slow down the progression of the disease with fewer side effects.”

Side effects indeed are a major concern with treatments for Duchenne and other types of muscle disorders.

Corticosteroids, which are a class of medications that work by mimicking the hormone cortisol, have long been part of standard-of-care treatment for DMD. These powerful anti-inflammatory medicines can help to lessen muscle damage, but their long-term use commonly causes problematic side effects like high blood pressure, weight gain, emotional disturbances, and weakened bones. When used in children, corticosteroids also can stunt growth.

Vamorolone is a dissociative corticosteroid — a newer type of therapy that aims to deliver anti-inflammatory effects similar to those seen with traditional corticosteroids, but with fewer side effects, due to differences in how the drug interacts with cortisol receptors in the body.

We hope this new treatment will minimize some of the side effects of steroids, further improving patient’s quality of life which is our main goal.

That’s important to patients, Magnano said.

“We hope this new treatment will minimize some of the side effects of steroids, further improving patient’s quality of life which is our main goal,” Magnano said.

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Santhera obtained the rights to vamorolone in 2020 from the therapy’s original developer ReveraGen BioPharma. Just a few months ago, Santhera closed a deal with Catalyst Pharmaceuticals giving Catalyst the rights to market vamorolone in the U.S., Canada, and Mexico.

A decision from the U.S. Food and Drug Administration (FDA) on vamorolone for DMD is expected next week.

Applications seeking approval of vamorolone in the U.S. and Europe are based on data from a clinical trial called VISION-DMD trial (NCT03439670). That study compared vamorolone against a conventional corticosteroid, specifically prednisone, in 121 boys with DMD.

The results showed that both treatments were similarly effective at preserving motor function compared with a placebo, and data at almost one year showed no loss of efficacy for patients who switched from prednisone to vamorolone.

Data from VISION-DMD also showed that, on average, patients given vamorolone maintained growth similar to what was seen for boys on placebo, whereas patients given prednisone, on average, experienced growth stunting. In patients who switched from prednisone to vamorolone, growth tended to improve after switching to the experimental therapy.

Markers of bone health tended to worsen with prednisone but not with vamorolone in VISION-DMD, data showed.

“The recommendation by the CHMP is a testament to the dedication and collaboration of all those involved in the development of vamorolone, including ReveraGen Biopharma, the DMD patient community, researchers, healthcare professionals, and our employees,” Eklund said.

ReveraGen is sponsoring an ongoing Phase 2 study (NCT05166109) testing vamorolone in men with Becker muscular dystrophy. The study is still recruiting participants, ages 18-64, at a site in Pittsburgh.