Genetic schreef op 18 mei 2021 13:22:
Voor de mensen die informatie willen (ook over de concurrentie) want dat verklaart de tegenvaller die dhr. de Vries altijd onder het tapijt probeert te schuiven. Motley Fool
This company focuses on developing new small-molecule drugs that treat rare diseases caused by faulty enzymes. BioCryst made the transition to a commercial-stage business last December when the Food and Drug Administration approved its first drug, Orladeyo, to prevent hereditary angioedema (HAE) attacks.
Bradykinin is an immune-system protein that performs many functions, including dilation of tiny blood vessels at the site of an injury. Production of bradykinin is largely unregulated in HAE patients, which leads to painful, uncontrolled swelling for no good reason. Orladeyo is a once-daily capsule that prevents another enzyme, kallikrein, from producing excess bradykinin.
Sales of Orladeyo got off to an encouraging start, producing $11 million in top-line revenue for BioCryst during the first three months of 2021; recent approvals in Europe should accelerate sales before the year's finished.
BioCryst's late-stage development pipeline also includes an orally available treatment for paroxysmal nocturnal hemoglobinuria (PNH) called BCX9930. In the second half of 2021, the company will begin pivotal studies that could make the drug the first needle-free treatment option for this life-threatening blood disorder.
11 mio in de states en introductie in EU met betere toedieningsvorm . De taart word wel kleiner