FDA Approves Novartis’ Tasigna for Children with Rare Leukemia
March 26, 2018
Rare Daily Staff
The U.S. Food and Drug Administration expanded the approved use of Tasigna to include treatment of first- and second-line pediatric patients one year of age or older with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase.
Tasigna is was also approved for and children with the condition who are resistant or intolerant to prior tyrosine kinase inhibitor therapy.
CML is a type of blood cancer where the body produces malignant white blood cells. Almost all patients with CML have an abnormality known as the “Philadelphia chromosome,” which produces a protein called BCR-ABL. This protein helps fuel the proliferation of malignant white blood cells in affected patients.
The FDA granted the new indications under its Priority Review designation. The FDA based its approval on two studies evaluating the efficacy and safety of Tasigna in pediatric patients two years to less than 18 years of age with Philadelphia chromosome-positive chronic myeloid leukemia.
Adverse reactions in the pediatric studies were generally consistent with those observed in adults, except for laboratory abnormalities of hyperbilirubinemia, which were reported at a higher frequency than in adult patients. The long-term effects of prolonged treatment with Tasigna in pediatric patients is unknown.
In the United States, Tasigna is now indicated for the treatment of adult and pediatric patients one year of age or older with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia. Tasigna is also indicated for the treatment of pediatric patients one year of age or older with Philadelphia chromosome-positive chronic myeloid leukemia resistant or intolerant to prior tyrosine kinase inhibitor therapy, as well as adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase and accelerated phase, resistant or intolerant to prior therapy that included imatinib.
March 26, 2018
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