MedPath

FDA Approves Asciminib (Scemblix) for Newly Diagnosed Philadelphia Chromosome-Positive CML

• The FDA has granted accelerated approval to asciminib (Scemblix) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase. • The approval was based on the ASC4FIRST trial, a randomized, active-controlled, open-label study that compared asciminib to investigator's choice of tyrosine kinase inhibitor (TKI). • Asciminib demonstrated a significantly higher major molecular response (MMR) rate at 48 weeks compared to TKIs (68% vs. 49%, p < 0.001) in the ASC4FIRST trial. • Common adverse effects associated with asciminib included musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea.

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to asciminib (Scemblix) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in the chronic phase. This approval marks a significant advancement in the treatment landscape for CML, offering a new first-line option for patients.
The approval was primarily based on the results of the ASC4FIRST trial (NCT04971226), a multicenter, randomized, active-controlled, open-label study. In this trial, 405 patients were randomized in a 1:1 ratio to receive either asciminib or the investigator’s choice of a selected tyrosine kinase inhibitor (TKI), which could be imatinib (Gleevec), nilotinib (Tasigna), dasatinib (Sprycel), or bosutinib (Bosulif).

Superior Efficacy of Asciminib

The primary efficacy outcome of the ASC4FIRST trial was major molecular response (MMR) at 48 weeks. Asciminib demonstrated a statistically significant and clinically meaningful improvement in MMR rates compared to TKIs. Specifically, the MMR rate in the asciminib arm was 68% (95% CI, 61-74), while the TKI arm achieved an MMR rate of 49% (95% CI, 42-56), with a p-value of less than 0.001. Further analysis revealed MMR rates of 69% (95% CI, 59-78) and 40% (95% CI, 31-50) in the asciminib and TKI groups, respectively (p-value <0.001).

Safety Profile

The safety profile of asciminib was evaluated in a pooled population that included patients with both newly diagnosed and previously treated Ph+ CML in the chronic phase. The most common adverse effects, occurring in 20% or more of patients, were musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea. Common laboratory abnormalities observed in 40% or more of patients included decreased lymphocyte count, decreased leukocyte count, decreased platelet count, decreased neutrophil count, and decreased corrected calcium.

Dosing and Administration

The FDA recommends a dosing regimen of asciminib at 80 mg once per day or 40 mg twice per day, administered at approximately 12-hour intervals.
This approval provides a valuable new option for patients newly diagnosed with Ph+ CML, potentially improving treatment outcomes and quality of life.
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 Clinical Trials

Related Topics

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 Asciminib as First-Line Treatment for Chronic Myeloid Leukemia

The FDA has granted accelerated approval to asciminib (Scemblix) as a first-line treatment for adults with Philadelphia chromosome-positive chronic myeloid leukemia (CML).
Asciminib, a tyrosine kinase inhibitor, offers a novel mechanism of action by blocking the BCR-ABL protein at a unique site, potentially improving efficacy and tolerability.

FDA Grants Accelerated Approval to Asciminib for Newly Diagnosed Chronic Myeloid Leukemia

The FDA has granted accelerated approval to asciminib for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase.
The approval was based on Phase 3 trial data involving 405 patients, demonstrating a major molecular response rate of 68% at 48 weeks with asciminib.

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 Asciminib (Scemblix) for Newly Diagnosed Chronic Myeloid Leukemia

The FDA has granted accelerated approval to asciminib for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (CML) in chronic phase.
Asciminib, a first-in-class STAMP inhibitor, was approved based on the ASC4First trial results, which demonstrated superior major molecular response (MMR) rates at 48 weeks compared to TKIs.

FDA Approves Asciminib (Scemblix) for Newly Diagnosed Philadelphia Chromosome-Positive CML

The FDA granted accelerated approval to asciminib (Scemblix) for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (CML) in chronic phase.
The approval was based on the phase 3 ASC4FIRST trial, which compared asciminib to investigator-selected tyrosine kinase inhibitors (TKIs) in patients with Ph-positive CML.

FDA Approves Asciminib (Scemblix) as First-Line Treatment for Ph+ Chronic Myeloid Leukemia

The FDA granted accelerated approval to asciminib for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia.
The approval was based on the ASC4FIRST trial, which demonstrated a significantly higher major molecular response rate at 48 weeks compared to standard TKIs.

FDA Approves Asciminib (Scemblix) as First-Line Treatment for Ph+ CML-CP

The FDA granted accelerated approval to 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 existing TKIs.

FDA Grants Accelerated Approval to Asciminib for Newly Diagnosed Chronic Myeloid Leukemia

The FDA has granted accelerated approval to asciminib (Scemblix) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML).
Asciminib, a BCR::ABL1 kinase inhibitor, showed a major molecular response (MMR) rate of 68% at 48 weeks in the ASC4FIRST trial.

FDA Approves Asciminib for Newly Diagnosed Philadelphia Chromosome-Positive CML

The FDA granted accelerated approval to asciminib for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase.
Asciminib demonstrated a major molecular response (MMR) rate of 68% at 48 weeks, compared to 49% in patients treated with standard tyrosine kinase inhibitors (TKIs).

Reference News

[1]
Asciminib Gets Accelerated Approval for Ph+ CML - Oncology Nursing News
oncnursingnews.com · Oct 29, 2024

The FDA granted accelerated approval to asciminib (Scemblix) for newly diagnosed Ph+ CML in chronic phase, based on the ...

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