Novartis Gains First-Line CML Approval for Scemblix, Eyes $3B Peak Sales

The FDA has granted accelerated approval to Novartis’ Scemblix for the treatment of adults newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia (CML) in the chronic phase. This expansion into the first-line setting significantly broadens the eligible patient population by approximately four times.

This approval was achieved through the FDA’s accelerated approval pathway, which requires Novartis to confirm the clinical benefits of Scemblix in this specific indication via a follow-up study. Victor Bulto, U.S. President of Novartis, emphasized the importance of having treatment options that are highly effective and well-tolerated, suggesting that Scemblix meets these criteria and could significantly alter the treatment landscape for many CML patients.

Novartis projects that this first-line approval will help to meet its sales targets for Scemblix, a drug that acts as an oral tyrosine kinase inhibitor. CEO Vas Narasimhan has expressed confidence in the drug's future, projecting peak sales of over $3 billion and noting that it may be exempt from the Inflation Reduction Act’s drug price negotiations due to its targeting of a rare disease.

In the third quarter, Scemblix was a significant growth driver for Novartis, with sales surging 72% year-over-year at constant currencies, reaching $182 million. This performance exceeded analyst expectations by 3%, according to Jefferies analyst Peter Welford.

Scemblix works by inhibiting the BCR-ABL1 fusion protein, a hallmark of certain leukemias. First approved three years ago for CML, it initially received accelerated approval for treating Philadelphia chromosome-positive, chronic phase CML, and full approval for the same patient population with the T315I mutation.

The recent label expansion was supported by data from the Phase III ASC4FIRST study. The study demonstrated that 68% of patients treated with Scemblix achieved a major molecular response, compared to 49% of patients receiving standard tyrosine kinase inhibitors like imatinib and nilotinib. This 20 percentage point difference was statistically significant, with a p-value of less than 0.001.

Additionally, molecular responses with Scemblix were deeper, and the drug’s safety profile remained consistent with previous studies. This suggests that not only is Scemblix more effective, but it also maintains an acceptable safety profile, making it a promising option for treating newly diagnosed CML patients.