Novartis broadens Scemblix use for first-line CML patients

In a significant development, the FDA has announced that Novartis’ chronic myeloid leukemia (CML) drug, Scemblix, is now available for newly diagnosed patients. Originally approved in 2021 as a third-line treatment, the drug's approval has now been extended to first-line patients, addressing the need for more effective and tolerable treatment options early in the disease's progression.

Victor Bulto, President of Novartis’ US operations, emphasized the necessity for this advancement. He revealed that approximately half of newly diagnosed CML patients fail to achieve a target response within the first year of standard treatment; many either discontinue or switch their therapies. The FDA's accelerated approval will nearly quadruple the number of patients eligible for Scemblix, specifically those with Philadelphia chromosome-positive chronic myeloid leukemia, or Ph+ CML-CP.

Bulto highlighted this approval as a testament to Novartis' commitment to pushing the boundaries of patient care. The decision was grounded in Phase 3 clinical trial data, which demonstrated that Scemblix surpassed Novartis' first-generation drug Gleevec and other existing therapies chosen by investigators in achieving a major molecular response (MMR), a crucial measure of disease control. Additionally, patients on Scemblix experienced fewer adverse events compared to those on other medications.

With this new approval, Scemblix enters the competitive field of second-generation tyrosine kinase inhibitors (TKIs) which includes Bristol Myers Squibb’s Sprycel, Pfizer’s Bosulif, and Novartis’ own Tasigna, along with various generic alternatives. Despite this competition, Novartis CEO Vas Narasimhan had previously described Scemblix as a "wild card" drug, acknowledging both its high risk and high potential reward.

During Novartis' second-quarter earnings call, Narasimhan projected that Scemblix could achieve peak sales of over $3 billion. He also reminded investors that the drug enjoys patent protection until the mid-2030s. The challenge, as Bulto explained, lies in ensuring broad adoption of Scemblix by both patients and healthcare providers, especially given the availability of other generic therapies. However, Bulto expressed confidence in Novartis' strategic plan to achieve this goal.

This FDA approval represents a significant milestone in the treatment of chronic myeloid leukemia, offering newly diagnosed patients a promising new option that could improve their response rates and overall treatment experience. Novartis' ongoing commitment to innovation and patient care continues to drive advancements in the field, providing hope and better outcomes for those affected by this challenging disease.