Pfizer’s own R&D efforts in sickle cell disease have fallen short, so the pharmaceutical giant is turning to M&A to gain a presence in the blood disorder. The acquisition of Global Blood therapeutics brings the commercialized drug Oxbryta plus a pipeline of other therapeutic candidates.
In acquiring the two early-stage Sanofi drugs for sickle cell disease, Global Blood Therapeutics aims to complement Oxbryta, its FDA-approved treatment for the rare blood disorder. Meanwhile, Sanofi can turn its full sickle cell efforts on a gene-editing therapy partnered with Sangamo Therapeutics.
Health care professionals can refer patients to the best options for diagnostic imaging covered by Medicare to give more people the medical assistance they need without extra financial stress.
In an open letter published by BIO, the executives emphasized the importance of science-driven evaluation of medicines and regulatory independence. The letter came amid growing concern about political pressure over the FDA, as well as lack of diversity in trials and companies being too quick to announce data that are inconclusive.
The first FDA approval for a NASH drug, data on "off-the-shelf" CAR-T cells and a potential approval of a controversial drug for Alzheimer's disease are some of things in store for 2020.
The agency gave accelerated approval to GBT's Oxbryta (voxelotor) for sickle cell disease in patients aged 12 and older. The drug works by inhibiting sickle hemoglobin polymerization.
The agency granted the application for accelerated approval priority review, but is not requiring an advisory committee meeting. A confirmatory trial measuring the drug's ability to reduce stroke risk is planned for the fourth quarter.
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The company presented updated data for the drug, voxelotor, at the European Hematology Association conference in Amsterdam Friday.