Drugs Medical Pharma

FDA approves Santhera’s Agamree for Duchenne muscular dystrophy

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October 31, 2023: The US Food and Drug Administration (FDA) has given the green light to Santhera Pharmaceuticals’ drug, Agamree (vamorolone), for treating Duchenne muscular dystrophy (DMD).

This approval not only brings hope to the DMD community but also promises financial gains, with Catalyst Pharmaceuticals, the holder of Aganree’s North American marketing rights, poised to receive $36 million in licensing payments. Moreover, Santhera, the Switzerland-based Pharma company, stands to gain up to $105 million in sales and a share of royalties.

Out of this total payment, $26 million is earmarked to fulfill Santhera’s third-party milestone obligations, a part of the arrangement made when the company acquired the drug from Idorsia and ReveraGen BioPharma for an approximate sum of $17.9 million in 2020. Importantly, Catalyst Pharmaceuticals will also assume responsibility for Santhera’s third-party royalty obligations on Agamree’s North American sales across all indications.

Expected launch 

With the FDA’s approval, Agamree is now on track for its US launch, which is expected in the first quarter of 2024.

DMD: A Rare Genetic Disorder

Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle degeneration and weakness. It impacts an estimated six individuals in every 100,000 in Europe and North America, according to the Muscular Dystrophy Association.

The FDA’s approval of Agamree relies on data from the Phase IIb trial (NCT03439670) and safety information gathered from three open-label studies. Notably, Agamree received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) in October, indicating a strong likelihood of approval in the European Union.

Year of breakthroughs

In addition to Agamree, the year has seen another groundbreaking development in the DMD landscape. Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl) secured approval as the first gene therapy for DMD, an important milestone in the treatment of this condition.

The FDA’s approval of Agamree offers hope to those affected by this rare genetic disorder and highlights the promise of innovative treatments in the field of rare diseases.

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