The FDA approved AstraZeneca drug Voydeya as an add-on to standard therapies for paroxysmal nocturnal hemoglobinuria, a rare blood disorder. Alexion, now AstraZeneca’s rare disease subsidiary, added the small molecule to its pipeline via a 2019 acquisition.
AstraZeneca dominates the market for drugs that treat rare blood disorder paroxysmal nocturnal hemoglobinuria. Roche drug crovalimab met the two main goals of its pivotal test, and if approved, would offer a dosing edge compared to AstraZeneca’s two blockbuster PNH medicines.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
Nearly all patients treated with the Novartis drug iptacopan achieved the hemoglobin goals of a pivotal study compared to zero patients who received the AstraZeneca drugs that are the standard of care in a rare blood disorder. The results were presented Tuesday during the annual meeting of the American Society of Hematology.
The FDA approved Apellis Pharmaceuticals drug pegcetacoplan (Empaveli) as a treatment for a rare blood disorder that is currently treated with medicines sold by Alexion Pharmaceuticals, a biotech being acquired by AstraZeneca. But Apellis’s drug beat Alexion drug Soliris in a head-to-head test, and the smaller company aims to steal market share from its larger rival.
The companies will use a combination of more than seven years of medical record data with five years of prospective data on 5,000 MS patients, including real-world outcomes data and MRI images. Other disease areas to be explored include hematology and rare diseases.
Soliris, the drugmaker's cash cow approved for the same disease, faces biosimilar competition as patent expiry looms.
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.