Billed as potential blockbuster, Novartis' sickle cell drug scores FDA's breakthrough therapy status
Touted by Novartis chief Vas Narasimhan as a potential blockbuster, the Swiss drugmaker’s sickle cell disease (SCD) drug has secured the FDA’s breakthrough therapy designation for its ability to prevent debilitatingly painful vaso-occlusive crisis for patients affected by the group of inherited red blood cell disorders that typically afflict those of African ancestry.
SCD patients have atypical hemoglobin molecules, which can distort red blood cells into a sickle, or crescent, shape. Symptoms such as anemia, repeated infections and periodic episodes of pain called vaso-occlusive crises (VOC) when sickle shaped red blood cells get stuck inside blood vessels begin to appear in early childhood. These episodes deprive the body of oxygen-rich blood, which can culminate in widespread tissue and organ damage — particularly in the lungs, kidneys, spleen, heart and brain — and drastically diminish life expectancy.
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