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Pfizer’s Rare Disease late-stage pipeline currently includes three genetherapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. Inflammation and Immunology.
Meanwhile, BioMarin’s genetherapy Roctavian is approved by the FDA for the treatment of hemophilia A. Onureg (azacitidine), a leukemia therapy, also saw large growth of 70 percent to $124 million in 2022, and the multiple sclerosis drug Zeposia (ozanimod) generated $250 million in 2022, an 87 percent increase.
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