Rare Diseases

Zevra Therapeutics said it expects the therapy to be commercially available in the United States in 8 to 12 weeks.

The approval was based on data from the phase 3 MANDARA trial, where the therapy met the primary endpoint of proportion of patients in remission at weeks 36 and 48.

Results from the PALISADE study were published in The New England Journal of Medicine.

Ascendis Pharma said it is currently completing manufacturing of commercial product, which it anticipates will be available in the US in the first quarter of 2025.

Data will be discussed with regulatory authorities “in the coming months.”

The approval of eculizumab-aagh offers more treatment options for patients with rare diseases PNH and aHUS.

The drug was FDA approved as antihemophilic factor (recombinant), Fc-VWF-XTEN Fusion Protein-ehtl (Altuviiio) in February 2023.

Eculizamab-aeeb is the 53rd FDA-approved biosimilar in the US.

WHIM syndrome is characterized by low levels of neutrophils and lymphocytes in the blood due to dysfunction in the CXCR4 receptor, which results in serious and frequent infections.

Fidanacogene elaparvovec-dzkt (Beqvez) is a 1 time treatment that allows patients with hemophilia B to produce factor IX themselves.

The company’s investigational ABCWY vaccine candidate will be reviewed by the FDA by February 14, 2025.