Novartis' Scemblix has been granted accelerated approval from the FDA for adults with newly diagnosed chronic myeloid leukaemia.
The oral tyrosine kinase inhibitor (TKI) is suitable for patients with Philadelphia chromosome-positive CML in the chronic phase.
Approval was accelerated by the FDA owing to the superior efficacy of Scemblix compared with all the standard of care therapies currently available for CML.
Scemblix has also demonstrated its favourable safety and tolerability profile during Phase III trials.
Patient eligibility is also enhanced compared to current standard of care medications, as both newly diagnosed and previously treated adults are able to take it.
During the Phase III ASC4FIRST trial, Scemblix:
- Demonstrated a superior molecular response rate (MMR) to all investigator-selected TKIs and imatinib alone.
- Was the first treatment to demonstrate superior efficacy, along with a favourable safety and tolerability rate compared to imatinib and second-gen TKIs
- Initiated deeper rates of molecular responses compared with imatinib-TKIs and imatinib alone
- Demonstrated efficacy in newly diagnosed patients
While TKIs have transformed CML into a chronic disease, efficacy and safety challenges continue to hinder the long-term treatment success of patients.
Currently, nearly half of CML patients don't meet efficacy milestones (MMR) and almost a quarter of patients discontinue or switch treatment within one year.
“While there are a range of effective TKIs currently available for newly diagnosed patients, clinicians frequently have had to weigh sacrificing either efficacy or tolerability,” said Jorge Cortes, Director, Georgia Cancer Center.
“In the first-of-its-kind ASC4FIRST trial, Scemblix achieved impressive results across all three parameters of efficacy, safety and tolerability versus all standard of care TKIs. This Scemblix data has the potential to be practice-changing.”
Chief Scientific Officer of the Leukemia & Lymphoma Society, Lee Greenberger, added: “Many patients who are newly diagnosed with CML struggle to navigate this chronic condition and may switch or even stop treatment because of side effects that interrupt their daily lives,”
“That’s why approvals of new first-line treatment options are so important. For patients, finding a medicine that’s right for them at the very beginning of treatment may lead to better long-term disease control with fewer side effects.”
Novartis' President (US) Victor Bulto, iterated: “We are proud to help redefine CML treatment once again with Scemblix, as we continue to deliver on our 20+ year commitment to innovation and support in CML,”
“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 as they manage chronic conditions. With this approval, we can offer newly diagnosed adult Ph+ CML-CP patients a new treatment option that combines both, with the potential to change the trajectory of many more people living with CML.”
References
1 Kota VK, Wei D, Yang D, et al. Treatment Patterns and Modifications of Tyrosine Kinase Inhibitors (TKI) Therapy in Early Lines in Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP): Real-World Analysis from a Large Commercial Claims Database in the United States (US). Blood. 2023;142:5190.
2 Hochhaus, A., & Ernst, T. 2021. TKI discontinuation in CML: How do we make more patients eligible? How do we increase the chances of a successful treatment-free remission? Hematology Am Soc Hematol Educ Program, 2021(1), 106–112.