Novartis Achieves Dual Approval in China for Asciminib and Ribociclib on the Same Day, Marking Major Advances in Leukemia and Breast Cancer Treatment

May 16, 2025 21:05 CST Updated 21:05

Novartis

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Text by Xiao Wei

On May 14, a new leukemia drug and a new indication for a tumor product from Novartis were approved by the National Medical Products Administration (NMPA) on the same day.

The newly approved drug is Scemblix (Asciminib Tablets), used to treat adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in the chronic phase (CP). It is the world's first and currently the only approved targeted drug based on the ABL myristoyl pocket (STAMP) mechanism.

Novartis' CDK4/6 inhibitorKaililong (Ribociclib Succinate Tablets)Then it was approvedIn combination with aromatase inhibitors, as adjuvant therapy for patients with early breast cancer who are hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative, and at high risk of recurrence.AllThe world's first and currently only approved CDK4/6 inhibitor for adjuvant treatment of broad-stage II-III HR+/HER2- breast cancer。

Asciminib andRibociclib, are two star products of Novartis. Novartis focuses on leukemia and breast cancer in these twoOncology TrackHas always been deeply involved and invested in,AndBoth have multiple iterative products on the market, and the promotion of these two approved products will be important focal points for Novartis in these two fields.

Breast Cancer: From First-Line Treatment in Advanced Stages to Adjuvant Therapy in Early Stages

The approval of Ribociclib was based on the NATALEE study.The research results show that,Compared with单纯辅助内分泌治疗,瑞波西利辅助治疗强化，The 4-year risk of breast cancer recurrence and distant metastasis both decreased by 28.5%, with consistent benefits observed across all subgroups, including those without lymph node metastasis or with stage II disease.，Continuous Reduction in Mortality Risk: 4-Year Follow-Up DataDisplayRibociclib has good long-term tolerability.

Breast cancer is the second most common malignant tumor in women, among which HR+/HER2- early breast cancer can beCure, butApproximately one-third of patients receiving conventional adjuvant endocrine therapy will still experience recurrence and metastasis, progressing to an incurable condition. Recurrence represents a clear unmet need in the treatment of early breast cancer.

RibociclibIs the second approved globallyCDK4/6 Inhibitor, Approved in the United States in March 2017 for the treatment of advanced breast cancer. In the first-line treatment of advanced breast cancer,RibociclibThe total survival period is 63.9 months, demonstrating significant advantages. In 2024, the FDA approved ribociclib combined with aromatase inhibitors for adjuvant treatment of HR+/HER2- early-stage breast cancer in stages II and III, including patients without lymph node metastasis (N0).Expanded to the field of adjuvant treatment for early breast cancer.

Ribociclib 2023JanuaryObtainNMPA Approval in ChinaLaunched as the initial endocrine therapy for premenopausal or perimenopausal female patients with HR+/HER2- locally advanced or metastatic breast cancer.A few months later, againApproved for First-Line Treatment of HR+/HER2- Advanced Breast Cancer in Postmenopausal Women.

Currently, Ribociclib has been approved in more than 100 countries and regions worldwide.NCCNRecommend ribociclib adjuvant therapy as a Class 1 preferred treatment option for HR+/HER2- early breast cancer patients at high risk of recurrence.

CML: Better Options After TKI Resistance

Asciminib ApprovedBased on the pivotal Phase III ASC4FIRST trial data。ASC4FIRSTIt is the first Phase III trial to conduct a randomized head-to-head comparison with approved standard treatments, namely first- and second-generation tyrosine kinase inhibitors (TKIs).

Efficacy data show that, compared with first- or second-generation TKI regimens, the proportion of patients treated with Asciminib who achieved MMR (i.e., BCR::ABL1 fusion gene level ≤0.1%) at week 96 significantly increased versus the control group (74.1% vs 52.0%). In terms of the response rate, the median time to achieve MMR was 24 weeks for patients treated with Asciminib, which was 12–24 weeks shorter than the control group. Meanwhile, the DMR achievement rate was twice that of the control group.

In terms of safety, compared with the first and second generation TKIs, the incidence of adverse events greater than Grade 3 with Asciminib was lower, and the risk of treatment discontinuation due to adverse events was reduced by more than half. Due to better tolerability, nearly 80% of patients receiving Asciminib remained on first-line treatment benefit over the 96-week observation period, while only 60% in the control group continued with first-line treatment.

Asciminib is a novel allosteric inhibitor targeting the BCR-ABL1 kinase. Unlike traditional TKIs, it binds to the myristoyl pocket (STAMP) of the target protein and inhibits the target protein by altering its conformation. Asciminib demonstrates higher selectivity and significantly reduces off-target effects.

The launch of Imatinib ushered in the era of targeted therapy for CML, and subsequently,TKIs has changed the treatment landscape for CML patients, bringing CML into an era of being treatable and controllable as a chronic disease, but there are still some patients for whomTKI ResistanceOrIntolerance, Asciminib is this typeCMLA new treatment option for patients, and Asciminib demonstrates superior performance in both efficacy and safety.。In the updated CML treatment guidelines from the National Comprehensive Cancer Network (NCCN), asciminib is recommended as the first-line treatment for newly diagnosed Ph+ CML-CP.

AsciminibApproved for marketing in the United States in October 2021, for the treatment ofPreviously received twoAndThe above-mentioned TKIsTreatment of Ph+CML-CPAdult patients, and in 2024 it was approved for first-lineTreatment of adult patients with newly diagnosed Ph+ CML-CP.ApprovedFirst-line treatmentAfter, the patient population for Asciminib has expanded by approximately 4 times.. 2022,AsciminibSuccessively approved for entry into Japan and the European Union, used forPatients with Ph+CML-CP treated with TKIs。

According to incomplete statistics,EyeFrontAsciminib DevelopmentFinished5Phase III clinical trials, allFor CML, the treatment lines span acrossOneLine, Second Line,ThreeLine, Maintenance/Consolidation Therapy。AsciminibIt is one of the main drivers of Novartis' current performance growth, and Novartis adopts a full-line coverage development strategy for this product.

Source of this article: Pharma Innovation

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