The European Commission (EC) has approved Santhera Pharmaceuticals’ Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients aged four years and older.
Estimated to affect one in every 3,500 male births worldwide, DMD is a rare muscle-wasting disorder caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.
Over time, this causes progressive loss of muscle strength, with most patients requiring full-time use of a wheelchair by their early teens. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure.
Agamree is a dissociative steroid that works in a similar way to existing corticosteroids, the current standard of care for children and adolescent patients with DMD, but without the same safety concerns.
The EC’s decision on the drug, which follows a recent recommendation from the European Medicines Agency’s human medicines committee, was supported by results from the pivotal VISION-DMD study and three open-label studies.
In VISION-DMD, patients treated with Agamree on average maintained growth similar to those treated with placebo, while those treated with the corticosteroid prednisone on average experienced growth stunting. Additionally, those who switched from prednisone to Agamree after 24 weeks were, on average, able to resume growing in height over the remainder of the study.
Unlike corticosteroids, Agamree did not result in a reduction of bone metabolism or bone mineralisation in the spine after 48 weeks in the clinical studies, Santhera said.
Dario Eklund, chief executive officer of Santhera, said: “We are thrilled to receive approval from the EC to bring Agamree to patients in the EU, which highlights the favourable safety and tolerability profile over conventional corticosteroids, including benefits for bone health and growth.”
Eklund added that the company is “now focused on ensuring Agamree is made available to the Duchenne patients as soon as possible,” with a first commercial launch planned for Germany in the first quarter of 2024.
The EC’s decision comes less than two months after Agamree was approved by the US Food and Drug Administration to treat DMD in patients aged two years and older.
An exclusive license from ReveraGen for all indications worldwide to Agamree is held by Santhera, which has out-licensed rights to the drug for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics.