Pfizer In Pole Position To Benefit From Novartis Sickle Cell Drug Setback
Adakveo Fails A Phase III Study
Pfizer joined Novartis as the market leader in sickle cell disease when it acquired Global Blood Therapeutics last year. The US giant's Oxbryta may now move ahead following a clinical and regulatory setback for the Swiss major's Adakveo.
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The recent ASH meeting began with US FDA approvals of the first genetic medicines for sickle cell disease, but doctors and biopharma companies still see a need for new oral therapies and other options.
According to a study presented at the American Society of Hematology, based on existing gene therapy prices, equitable use of sickle cell disease gene therapy requires expanding into patients who are younger and have less severe disease.
The first gene-edited therapy will be submitted for sickle cell disease and transfusion-dependent beta thalassemia; it will compete with bluebird in both indications, but analysts anticipate blockbuster sales in excess of $2bn.