Scemblix was granted accelerated approval by the FDA for the treatment of certain patients newly diagnosed with chronic myeloid leukemia. The expanded indication increases the eligible patient population by approximately four times, according to Novartis.
The FDA on Tuesday approved the expansion of Novartis’ Scemblix for the treatment of adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase.
Granted under the regulator’s accelerated approval pathway, Tuesday’s label expansion pushes Scemblix into the first-line setting in chronic myeloid leukemia (CML), allowing its use in patients with chronic phase disease positive for the Philadelphia chromosome. To retain this approval, Novartis will need to verify the clinical benefit of Scemblix in this indication through a confirmatory study.
“Despite many advances in the field, patients still need treatment options that are highly effective with a favorable tolerability profile to help enable them to achieve meaningful outcomes,” Victor Bulto, U.S. president of Novartis, said in a statement. Scemblix fulfills both criteria and possesses the “potential to change the trajectory of many more people living with CML,” Bulto said.
Novartis said the first-line approval broadens the patient population for Scemblix by approximately four times, which will help the pharma achieve its sales target for its oral tyrosine kinase blocker. During the company’s second-quarter earnings call in July, CEO Vas Narasimhan projected “$3 billion-plus peak sales” for Scemblix, adding that it is likely to be exempt from the Inflation Reduction Act’s drug price negotiations because it targets a rare disease.
“A really great profile, great medicine, very excited about its future,” Narasimhan said of Scemblix during the investor call.
In Q3, Scemblix was one of Novartis’ main growth drivers, surging 72% year-over-year at constant currencies, bringing in $182 million. Scemblix’s sales in the quarter beat the analyst consensus by 3%, according to Jefferies analyst Peter Welford.
Scemblix is an orally available tyrosine kinase inhibitor that works by blocking the BCR-ABL1 fusion protein, a known hallmark of certain leukemias. The drug was first approved for CML three years ago, winning two indications. The first is for the treatment of Philadelphia chromosome-positive, chronic phase CML, for which the drug was given accelerated approval. The second is a full approval for the same patient population, with the additional specification that they carry the T315I mutation.
Tuesday’s label expansion was backed by data from the Phase III ASC4FIRST study, which showed that 68% of patients on Scemblix achieved major molecular response, compared with 49% of those treated with standard-of-care tyrosine kinase inhibitors such as imatinib and nilotinib. The difference of nearly 20 percentage points was statistically significant with a p-value of less than 0.001, according to Novartis.
Molecular responses were likewise deeper with Scemblix, while the drug’s safety profile remained consistent with what had been established in prior studies.
