Agamree approved in UK for DMD patients, 4 and older

Agamree expected to be available in UK in second half of 2024

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

A person wearing a baseball cap speaks into a cone-shaped megaphone.

Agamree (vamorolone) has been approved in the U.K. for treating Duchenne muscular dystrophy (DMD) in patients 4 and older.

This approval by the Medicines and Healthcare products Regulatory Agency (MHRA) follows a similar decision in the European Union. In the U.S., Agamree is approved for patients 2 and older.

“We are working towards making AGAMREE available to patients in the UK in the second half-year 2024, after NICE completes its pricing review. Initial European launch will be in Germany,” Shabir Hasham, MD, chief medical officer of Santhera Pharmaceuticals, which markets the therapy, said in a press release.

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“We are delighted that the first drug designed specifically for everyone with Duchenne has been approved in the UK,” said Emily Reuben, CEO and co-founder of Duchenne UK, and Alex Johnson, also a co-founder. “Duchenne UK and our partner charities, Joining Jack and the Duchenne Research Fund, funded the early-stage clinical research to test vamorolone in patients, when no-one else would. For it now to be available in the UK to treat DMD is proof that we can find better treatments for Duchenne and change things for Duchenne.”

The move makes Amagree the first therapy to be formally authorized as a treatment for DMD in the U.S., EU, and the U.K.

“We are delighted to have secured a third approval for AGAMREE to treat Duchenne from a major regulatory agency … within a couple months,” Hasham said.

Corticosteroids have for long been the treatment of choice for DMD due to its powerful anti-inflammatory properties. While corticosteroids don’t target the root cause of DMD, they slow disease progression by lessening muscle damage. The caveat is the wide range of potentially serious side effects with long-term corticosteroids. Among these are weight gain, fragile bones, stunted growth, and mood problems.

Agamree is a dissociative corticosteroid, meaning it provides similar clinical benefits with fewer side effects. Originally developed by ReveraGen BioPharma, which has been acquired by Santhera, the therapy was designed to bind more weakly than standard corticosteroids to glucocorticoid receptors, proteins that promote the activation of genes linked to corticosteroid side effects.

Also, contrary to most corticosteroids, no interaction occurs between Agamree and 11-beta HSD, an enzyme that enhances effects in the muscle, bone, brain, and kidneys.

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Approval based on data from 4 clinical trials

Agamree’s approval was based on data from four clinical studies, including the VISION-DMD trial (NCT03439670), which compared the efficacy and safety of Agamree against a conventional corticosteroid, specifically prednisone, in 121 boys with DMD.

Results showed the two therapies were comparable at preserving muscle function, as DMD patients who switched from prednisone to Amagree did not experience reduced treatment efficacy.

Additionally, boys on Amagree also tended to experience fewer side effects. Unlike prednisone, treatment with Agamree was not associated with delayed growth.

“In addition to its anti-inflammatory efficacy, both the [European Medicines Agency] and the MHRA recognize the benefits of treatment with Agamree for bone health and growth, underlining the favorable safety and tolerability profile of this novel medicine compared to conventional corticosteroids,” Hasham said.

Santhera will continue to assess the long-term effectiveness and safety of Agamree, the company noted.