Novartis Secures FDA Approval for Scemblix as First-Line Treatment for Newly Diagnosed CML, Anticipates $3B in Peak Sales

FDA Approval:
Novartis has secured FDA approval for Scemblix (asciminib) as a first-line treatment for newly diagnosed adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).

ASC4FIRST Trial:
The approval is based on the Phase III ASC4FIRST trial, which demonstrated superior major molecular response (MMR) rates at week 48 compared to investigator-selected standard-of-care tyrosine kinase inhibitors (TKIs) and imatinib alone.

Efficacy and Safety:
Scemblix showed significantly better efficacy with a favorable safety and tolerability profile, including fewer grade ≥3 adverse events, dose adjustments, and half the rate of adverse events leading to treatment discontinuation compared to imatinib and 2G TKIs.

Market Impact:
Novartis projects that Scemblix could reach peak sales of $3 billion, expanding its market share in the CML treatment landscape.

Mechanism of Action:
Scemblix is the first CML treatment that works by Specifically Targeting the ABL Myristoyl Pocket (STAMP inhibitor), offering a new mechanism of action compared to traditional TKIs.

Approval History:
Scemblix was previously granted FDA Breakthrough Therapy designation and was reviewed under the FDA’s Real-Time Oncology Review program, highlighting its potential to address a critical gap in CML management.