Santhera: FDA Approval for treatment of Duchenne muscular dystrophy

• Food and Drug Administration (FDA) approved AGAMREE (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in children and adults aged 2 years and older
• U.S. license holder Catalyst Pharmaceuticals plans for commercial launch in Q1-2024
• FDA approval triggers USD 36 million payment obligation from Catalyst to Santhera, of which USD 26 million will be used to cover Santhera’s third-party milestone obligations
• Following the recent positive CHMP opinion on AGAMREE in DMD, approval in the European Union by the European Commission (EC) is expected in late 2023
• AGAMREE would then become the first drug fully approved in both the U.S. and Europe for the treatment of patients with DMD

“We are delighted to secure FDA approval which comes just weeks after the positive opinion from the CHMP 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,” said Dario Eklund, CEO of Santhera. “Today is a very satisfying day for the Santhera team and we are grateful to the patients, their families and physicians who participated in the vamorolone program. We look forward to working closely with our partner Catalyst Pharmaceuticals as it prepares for U.S. commercialization of the product.”

“This new drug approval gives Duchenne patients and their families one more reason for hope,” said Pat Furlong, Founding President & CEO Parent Project Muscular Dystrophy (PPMD). “Steroids are considered standard of care for Duchenne, due to their valuable role in slowing disease progression. However, they often come with considerable side effects. Vamorolone has the potential to be an alternative steroid with a better tolerability profile addressing an important unmet medical need for patients.”

“I am excited for the Duchenne community who have been waiting a long time for an alternative to the current standard of care. Today’s news represents the culmination of many years of research to bring vamorolone to patients,” said Eric Hoffman, PhD, President and CEO of ReveraGen BioPharma. “We are pleased to have worked alongside Santhera, the DMD patient community, researchers and healthcare practitioners to reach this important milestone.”

The FDA approval of AGAMREE was based on the data from the pivotal Phase 2b VISION-DMD study as supplemented with safety information collected from three open-label studies, including extension studies. In these trials, AGAMREE was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. Compared with current standard of care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory and behavior. The studies in the development program were carried out by Santhera’s partner ReveraGen and 32 academic clinical trial centers in 11 countries.

With the FDA approval of AGAMREE for DMD, Catalyst will pay USD 36 million to Santhera, consisting of a USD 10 million approval milestone to the Company and an additional USD 26 million to cover contracted third-party milestone obligations. Furthermore, under the terms of the agreement, Catalyst will pay Santhera sales-based milestones of up to USD 105 million as well as up to low-teen percentage royalties, and will assume Santhera's corresponding third-party royalty obligations on vamorolone sales in all indications in North America.

Santhera will now transfer the U.S. marketing authorization (NDA) for AGAMREE to its partner Catalyst Pharmaceuticals who holds an exclusive license for AGAMREE in North America and plans to launch the product in the U.S. in Q1-2024.

In Europe, following the positive opinion from the CHMP received on October 12, 2023, approval of AGAMREE (vamorolone) for the treatment of DMD by the European Commission is expected in late 2023. Subject to approval, AGAMREE will be the only medicinal product fully approved in both territories, the EU and the U.S., for the treatment of DMD.

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