FDA approves Baxalta's longer-lasting hemophilia A drug
- The FDA has approved Baxalta's Adynovate (Antihemophilic Factor Recombinant, PEGylated) for use in patients with hemophilia A. The drug was modified to last longer in the blood and potentially require less frequent injections compared to unmodified Antihemophilic Factor.
- Adynovate is intended to be used on demand in order to reduce the frequency of bleeding episodes.
- Hemophilia A affects roughly 1 in 5,000 male births in the U.S. It is associated with bleeding episodes which affect the joints.
Adynovate is a powerful new treatment option for patients with hemophilia A, because it requires fewer and less frequent injections. It's approval stemmed from clinical data produced by a trial involving 137 adults and adolescents aged 12 and older. In the trial, conventional preventive therapy was compared to on-demand Adynovate therapy. Adynovate effectively reduced the number of bleeding episodes during routine care. Overall, Adynovate-treated patients experienced 95% fewer bleeding episodes, with 40% of patients reporting no bleeds at all.
With the approval of its first drug under the Baxalta name, this recently spun-off company has established expertise in hematology and immunology. Its portfolio currently grosses around $6 billion per year and CEO Ludwig Hantson has signaled Baxalta's intentions to launch 20 new products by 2020.
With this start, Baxalta has strengthened its position against Shire's continuing acquisition efforts. It appears unlikely that Baxalta will reconsider Shire's $30 billion offer in the near future given this latest success.