MedPath

FDA Approves Scemblix as First-Line Treatment for Chronic Myeloid Leukemia

• The FDA has granted accelerated approval to Novartis' Scemblix (asciminib) for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). • Scemblix, a STAMP inhibitor, demonstrated superior major molecular response rates compared to traditional tyrosine kinase inhibitors (TKIs) in the ASC4FIRST trial. • This approval expands the eligible patient population for Scemblix and offers a potentially safer and more effective alternative to existing first-line CML treatments. • Novartis is conducting further trials to confirm Scemblix's benefits, with potential for blockbuster sales and global market expansion.

The FDA has granted accelerated approval to Novartis' Scemblix (asciminib) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This decision, announced on Tuesday, marks a significant advancement in the treatment landscape for CML, offering a novel first-line option for patients.

ASC4FIRST Trial Results

The approval is based on data from the ASC4FIRST trial, which demonstrated that Scemblix achieved a significantly higher major molecular response (MMR) rate compared to traditional tyrosine kinase inhibitors (TKIs). At 48 weeks, 68% of patients treated with Scemblix achieved MMR, compared to 49% of those treated with TKIs. Furthermore, a deeper molecular response (DMR) was observed in 39% of patients on Scemblix versus 21% on TKIs.

Clinical Significance and Safety Profile

Scemblix, a first-in-class STAMP inhibitor, offers a distinct mechanism of action compared to traditional TKIs. The ASC4FIRST trial also suggested a favorable safety and tolerability profile for Scemblix, with fewer adverse events, treatment interruptions, and discontinuations compared to existing TKIs. This is particularly important as almost half of CML patients on TKIs may need to switch therapies within two years due to side effects or resistance.

Impact on CML Treatment

"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," said Lee Greenberger, chief executive of The Leukaemia & Lymphoma Society, emphasizing the importance of personalized treatment approaches in CML.

Current Treatment Landscape and Unmet Needs

While TKIs have dramatically improved survival rates in CML, with five-year survival rates increasing to around 70%, challenges remain. Current first-line treatments include generic imatinib and second-generation TKIs like dasatinib, nilotinib and bosutinib. However, the need for safer and more effective options persists, particularly for patients who experience intolerance or resistance to these therapies.

Ongoing Studies and Future Prospects

Novartis is required to provide further clinical evidence to confirm Scemblix's benefits as a first-line treatment. The ASC4FIRST trial is ongoing, with the next analysis scheduled at week 96. Additionally, the ASC2ESCALATE phase 2 trial, involving patients previously treated with a TKI, will contribute to the growing dataset supporting Scemblix's efficacy and safety.
Novartis anticipates that Scemblix could achieve blockbuster status, with potential annual sales of $3 billion. Submissions for approval in China and Japan have been completed, and a European submission is expected in 2025.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Novartis' 'Wild Card' Drug Scemblix Receives FDA Approval for Leukaemia Treatment

Novartis has secured FDA approval for Scemblix, a novel STAMP inhibitor, offering new hope for patients with previously-treated chronic myeloid leukaemia (CML). The drug has shown promising results in clinical trials, particularly for patients with the T315I mutation, and could significantly impact Novartis' CML portfolio.

FDA Grants Accelerated Approval to Novartis' Scemblix for Newly Diagnosed CML

The FDA has granted accelerated approval to Scemblix (asciminib) for adults with newly diagnosed Philadelphia chromosome-positive CML in the chronic phase (Ph+ CML-CP).
The approval expands Scemblix's eligible patient population by approximately four times, offering a new treatment option for those with CML.

FDA Approves Scemblix (asciminib) for Newly Diagnosed Chronic Myeloid Leukemia

The FDA has granted accelerated approval to Scemblix (asciminib) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
Asciminib is the first CML treatment that specifically targets the ABL myristoyl pocket, offering a novel mechanism of action compared to traditional tyrosine kinase inhibitors (TKIs).

FDA Approves Scemblix as First-Line Treatment for CML, Expanding Novartis' Reach

The FDA has approved Scemblix (asciminib) for first-line treatment of Philadelphia chromosome-positive chronic myeloid leukemia (CML) in the chronic phase.
This approval expands Scemblix's use to newly diagnosed CML patients, potentially quadrupling the treatable population for Novartis.

FDA Approves Novartis' Scemblix as First-Line Treatment for Chronic Myeloid Leukemia

The FDA granted accelerated approval to Scemblix (asciminib) for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia.
The approval was based on the Phase 3 ASC4FIRST trial, which demonstrated superior major molecular response rates at week 48 compared to standard tyrosine kinase inhibitors.

FDA Approves Novartis' Scemblix (asciminib) for First-Line CML Treatment

The FDA granted accelerated approval to Novartis' Scemblix (asciminib) for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP).
The approval was based on the ASC4FIRST Phase III trial, demonstrating Scemblix's superiority over standard-of-care tyrosine kinase inhibitors (TKIs) in achieving major molecular response (MMR) at week 48.

FDA Approves Novartis' Scemblix for Newly Diagnosed Chronic Myeloid Leukemia

The FDA granted accelerated approval to Scemblix (asciminib) for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (CML).
Scemblix, a STAMP inhibitor, demonstrated superior major molecular response (MMR) rates compared to standard tyrosine kinase inhibitors (TKIs) in the ASC4FIRST trial.

Scemblix Receives Accelerated FDA Approval for Newly Diagnosed Ph+ CML

The FDA granted accelerated approval to Scemblix (asciminib) for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
The approval was based on the ASC4FIRST Phase III trial, which demonstrated superior major molecular response (MMR) rates at week 48 compared to standard of care tyrosine kinase inhibitors (TKIs).

FDA Grants Accelerated Approval to Novartis' Scemblix for Newly Diagnosed CML

The FDA granted accelerated approval to Scemblix (asciminib) for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
Approval was based on the ASC4FIRST Phase III trial, which showed Scemblix had superior major molecular response (MMR) rates at week 48 compared to standard of care tyrosine kinase inhibitors (TKIs).

FDA Approves Novartis' Scemblix for Resistant Philadelphia Chromosome-Positive CML

The FDA has approved Scemblix (asciminib) for Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP) in adults after two or more tyrosine kinase inhibitors (TKIs).
Scemblix, a first-in-class ABL myristoyl pocket binder, offers a novel approach for patients with resistance or intolerance to existing TKI therapies.

Reference News

[1]
Novartis expands Scemblix label to first-line CML patients
endpts.com · Oct 29, 2024

FDA announced Novartis' Scemblix, a chronic myeloid leukemia drug, is now available for newly diagnosed patients, expand...

[2]
Novartis gets FDA okay for frontline use of Scemblix in CML - Pharmaphorum
pharmaphorum.com · Oct 30, 2024

Novartis secures FDA accelerated approval for Scemblix (asciminib) as first-line therapy for newly diagnosed Philadelphi...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy