Latest Rare Disease Treatments News

AFT Pharmaceuticals has received tentative US FDA approval for its Scomara cream to treat Facial Angiofibromas in Tuberous Sclerosis, but US commercialisation is postponed until 2029 due to orphan drug exclusivity. The approval bolsters regulatory prospects in other key markets where AFT anticipates competitive advantages.

AFT Pharmaceuticals secures tentative FDA approval for Scomara cream to treat rare facial tumours but faces a US launch delay due to orphan drug exclusivity. The approval bolsters prospects in other markets where AFT expects competitive advantages.

CLINUVEL Pharmaceuticals secures Canadian marketing approval for SCENESSE®, expanding access to the only approved systemic photoprotective treatment for erythropoietic protoporphyria (EPP).

Alterity Therapeutics has secured FDA alignment on the chemistry and manufacturing controls for its ATH434 Phase 3 trial in Multiple System Atrophy, marking a pivotal regulatory milestone ahead of the planned mid-2026 End-of-Phase 2 meeting.

Health Canada has requested additional information, extending its review of CLINUVEL’s SCENESSE® for erythropoietic protoporphyria (EPP) into 2026. Meanwhile, Canadian patients continue to access the treatment via a Special Access Program.

The European Medicines Agency has approved continuous bi-monthly dosing of CLINUVEL’s SCENESSE® for adult erythropoietic protoporphyria patients, removing previous annual dose limits and aligning Europe with US treatment standards.

Neuren Pharmaceuticals reports a 16% rise in Q2 2025 royalties driven by Acadia’s DAYBUE sales growth and expanding patient base in the US. Full-year sales guidance remains strong with European market entry on the horizon.

CSL has secured FDA approval for ANDEMBRY®, a pioneering treatment for hereditary angioedema, enabling an immediate US launch with promising clinical results. This milestone marks a significant advance in managing a rare but debilitating condition.

Neuren Pharmaceuticals reports a robust 11% rise in DAYBUE™ US net sales for Q1 2025, alongside a 17% jump in royalty income, setting the stage for an anticipated European market entry in early 2026.