FDA Grants Breakthrough Therapy Designation to Novartis' Scemblix® for First-Line CML
Novartis has announced that the FDA has granted Breakthrough Therapy designation to Scemblix® (asciminib) for treating adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
This marks the third Breakthrough Therapy designation for Scemblix.
- To qualify for Breakthrough Therapy designation, treatments must target serious or life-threatening conditions and show potential for substantial improvement over existing therapies on key clinical endpoints.
- Scemblix earned this designation based on positive results from the Phase III ASC4FIRST study, which showed superior major molecular response (MMR) rates at 48 weeks compared to several tyrosine kinase inhibitors (TKIs) including imatinib, nilotinib, dasatinib, and bosutinib, as well as compared to imatinib alone.
- Scemblix also demonstrated a favorable safety profile with fewer adverse events and lower treatment discontinuation rates compared to standard TKIs.
Currently, about half of newly diagnosed CML patients on standard TKIs do not achieve molecular response goals within a year, with many discontinuing or switching treatments due to intolerance.
The full results of the ASC4FIRST study will be presented at the ASCO conference on May 31. Additionally, Novartis will hold an investor event in Chicago on June 2 to explore the results and discuss the potential commercial prospects for Scemblix in first-line CML treatment, pending regulatory approval.
Novartis has now received 30 Breakthrough Therapy designations, highlighting its ongoing dedication to advancing medical innovation.
