paroxysmal nocturnal hemoglobinuria

With FDA approval, Apellis can challenge AstraZeneca in rare blood disorder

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 …

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Apellis’ Empaveli (pegcetacoplan) Receives the US FDA’s Approval for Paroxysmal Nocturnal Hemoglobinuria

Shots: The approval is based on P-III head-to-head PEGASUS study assessing Empavel vs Soliris (eculizumab) in 80 adults with PNH. Participants should be Soliris stable for at least 3mos. with a Hgb level of <10.5 g/dL at the screening visit Results: The study met its 1EPs demonstrating superiority to Soliris for the change from baseline …

Apellis’ Empaveli (pegcetacoplan) Receives the US FDA’s Approval for Paroxysmal Nocturnal Hemoglobinuria Read More »

Roche, PicnicHealth start RWD partnership with multiple sclerosis focus

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.