Catalyst Pharmaceuticals

Santhera Pharmaceuticals reports H1 2024 financial results, including CHF 14.1 million in revenue and a net loss of CHF 15.3 million. AGAMREE® (vamorolone) launched in Germany, Austria, and the U.S. for Duchenne muscular dystrophy (DMD), with strong market uptake. The drug received approval in the UK and NDA priority review in China. Santhera secured CHF 69 million in financing, expecting cash flow break-even by 2026.

Catalyst Pharmaceuticals announced FDA approval for AGAMREE® (vamorolone) to treat Duchenne Muscular Dystrophy (DMD) in patients aged two and older. AGAMREE, a novel corticosteroid with a favorable side effect profile, is expected to launch in Q1 2024. Catalyst holds exclusive North American rights and plans a patient assistance program to ensure access. AGAMREE showed comparable efficacy to standard treatments with fewer adverse effects in trials.

Catalyst Pharmaceuticals announced FDA approval for Santhera Pharmaceuticals' DMD treatment, Agamree, for patients aged two and older. The drug, similar to corticosteroids but with fewer side effects, is set for U.S. launch in Q1 2024. Catalyst plans a financial assistance program to enhance drug accessibility.

Catalyst Pharmaceuticals acquired North American commercial rights to vamorolone, a potential Duchenne muscular dystrophy (DMD) treatment, from Santhera Pharmaceuticals for $75M upfront plus $15M investment. Vamorolone, a dissociative corticosteroid, aims to offer DMD treatment benefits with fewer side effects. Regulatory decisions in the U.S. and Europe are pending, with a U.S. launch planned for early 2024 if approved.

Lore Wilkinson, a 91-year-old with a rare muscle disease, faces a $9,000 copayment for her medication, Firdapse, due to its orphan drug status granting Catalyst Pharmaceuticals exclusive market rights. This situation highlights the ongoing debate over the Orphan Drug Act, FDA's exclusivity policies, and their impact on drug prices and patient access.