Agamree approved in Europe to treat DMD boys ages 4 and older
Decision makes Duchenne therapy the 1st to be OK'd in both US and EU
The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects than standard steroid medications — for patients ages 4 and older with Duchenne muscular dystrophy (DMD).
The decision applies to all member states of the European Union, plus Iceland, Liechtenstein, and Norway.
The move makes Amagree the first therapy to be formally authorized as a treatment for DMD in both the U.S. and Europe, according to its developer Santhera Pharmaceuticals.
“We are thrilled to receive approval from the [European Commission] to bring AGAMREE to patients in the EU, which highlights the favorable safety and tolerability profile over conventional corticosteroids, including benefits for bone health and growth,” Dario Eklund, CEO of Santhera, said in a company press release.
Eklund added that the team at Santhera “is now focused on ensuring AGAMREE is made available to the Duchenne patients as soon as possible,” with plans to commercially launch the therapy in Germany early in the new year.
Therapy expected to have fewer side effects than standard corticosteroids
The approval of Amagree in Europe comes just a few months after a similar decision across the Atlantic by the U.S. Food and Drug Administration (FDA). The FDA approved Amagree in October to treat DMD patients ages 2 and older.
In the U.S. and elsewhere in North America, the therapy will be marketed by Catalyst Pharmaceuticals, which similarly expects commercial launch in the U.S. in early 2024.
The approvals of Amagree were applauded by the World Duchenne Organization, which counts many national patient groups, including Defeat Duchenne Canada, under its global umbrella.
“World Duchenne Organization is happy to see the development of AGAMREE, during which the involvement of many Duchenne patient organizations has been instrumental by providing early funding and contributing to clinical trial design, outcomes and recruitment, led to the first full market authorization in the European Union for treating Duchenne muscular dystrophy,” said Elizabeth Vroom, the organization’s chair.
Corticosteroids are powerful anti-inflammatory medications that have long been a mainstay of DMD treatment. While corticosteroids don’t directly affect the underlying cause of DMD, they can lessen muscle damage to help slow disease progression and preserve function. However, corticosteroids also are known for causing hard-to-manage side effects, including weight gain, fragile bones, stunted growth, and mood problems.
Agamree is a dissociative corticosteroid, which basically means that it is intended to provide similar clinical benefits with fewer side effects. Specifically, it binds more weakly than standard corticosteroids to glucocorticoid receptors, which lowers the activation of genes responsible for corticosteroid-associated side effects. Also, unlike most corticosteroids, Agamree does not interact with an enzyme called 11-beta HSD that amplifies effects in the muscle, bone, brain, and kidneys, thereby contributing to side effects.
World Duchenne Organization is happy to see the development of AGAMREE, during which the involvement of many Duchenne patient organizations has been instrumental by providing early funding and contributing to clinical trial design, outcomes and recruitment, led to the first full market authorization in the European Union for treating Duchenne muscular dystrophy.
Data from studies including the VISION-DMD clinical trial (NCT03439670) have suggested that Amagree is comparable to standard corticosteroids at preserving muscle function among patients. In that trial, individuals with DMD who switched from the corticosteroid prednisone to Amagree did not experience reduced efficacy.
Patients on Amagree also tended to experience fewer side effects, data have indicated. In particular, unlike prednisone, Agamree treatment was not associated with delayed growth.
Following the therapy’s U.S. approval, Santhera said it believes Amagree is poised to be the corticosteroid of choice for people with DMD.