The US Food and Drug Administration (FDA) has approved Santhera Pharmaceuticals’ Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients aged two years and older.
Santhera will now transfer the US marketing authorisation for Agamree to its partner Catalyst Pharmaceuticals, which holds an exclusive license for the drug in North America.
DMD is a rare muscle-wasting disorder estimated to affect one in every 3,500 male births worldwide. The disease is 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 in children and adolescent patients with DMD, but without the same safety concerns.
The FDA’s decision was supported by results from a phase 2b trial in which Agamree showed comparable efficacy to corticosteroids, with data suggesting a reduction in adverse events.
A final decision from the European Commission on Agamree is also expected later this year, following a recent recommendation from the European Medicines Agency’s human medicines committee.
Dario Eklund, Santhera’s chief executive officer, said: “We are delighted to secure FDA approval, which comes just weeks after the positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency.
“This is a hugely important moment for DMD patients who need an efficacious and well-tolerated therapy for this debilitating condition.”
Patrick McEnany, chairman and chief executive officer of Catalyst Pharmaceuticals, said: “We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with DMD and potentially other chronic inflammatory diseases.”
He added that the company will be introducing a “comprehensive financial assistance programme aimed at helping ensure accessibility and minimising patient co-pays and deductibles” to enhance the affordability of the drug.
The FDA approval of Agamree comes just a few months after the US regulator approved Sarepta Therapeutics’ DMD gene therapy.
The one-time treatment Elevidys (delandistrogene moxeparvovec-rokl) is specifically indicated for use in ambulatory patients aged between four and five years with a confirmed mutation in the DMD gene.