The US Food and Drug Administration (FDA) has approved Santhera Pharmaceuticals’ Agamree (vamorolone) for treating Duchenne muscular dystrophy (DMD).
The approval has triggered $36m in licensing payments from Catalyst Pharmaceuticals, which holds licensing rights for marketing Aganree in North America. According to the agreement, Santhera is also in line to receive up to $105m in sales-based milestones and a percentage of royalties.
The $26m from the total payment will cover Santhera’s third-party milestone obligations, as per the 27 October press release. The company acquired the drug from Idorsia and ReveraGen BioPharma for approximately $17.9m in 2020. Catalyst will also assume Santhera's third-party royalty obligations on Agamree North American sales in all indications.
The US launch of Agamree is planned for the first quarter of 2024. GlobalData forecasts the therapy to generate $216m in global sales in 2033.
GlobalData is the parent company of Pharmaceutical Technology.
DMD is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness. It affects six in 100,000 individuals in Europe and North America, as per the Muscular Dystrophy Association.
The FDA approval for Agamree is based on the Phase IIb trial (NCT03439670) along with the safety information from three open-label studies. In October, Agamree also received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for its approval in the EU.
The first gene therapy for the DMD, Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), was also approved this year.
In July, Santhera divested global Raxone (idebenone) programme to Chiesi Farmaceutici while still being eligible for milestone-based payments from the drug sales.
Another drug in Santhera’s pipeline, lonodelestat, is a selective peptide inhibitor of human neutrophil elastase, which has orphan drug designations in the EU and US for treating cystic fibrosis, alpha-1 antitrypsin deficiency (AATD), and primary ciliary dyskinesia (PCD).
