In which countries is Capmatinib approved?

Introduction to Capmatinib

What is Capmatinib?
Capmatinib (marketed under the trade name Tabrecta™) is an oral, small molecule mesenchymal‑epithelial transition (MET) inhibitor developed by Novartis Oncology under a license from Incyte Corporation. It was designed specifically to target MET, including the mutant variants produced by exon 14 skipping, which are known drivers of certain cancers such as non‑small cell lung cancer (NSCLC). Its development represents an effort to address a significant unmet medical need in patients whose tumors carry this mutation and who historically have had limited treatment options. The molecule is characterized by its selective binding to MET, resulting in the inhibition of downstream signaling pathways that promote tumor cell proliferation and survival.

Mechanism of Action
Capmatinib exerts its therapeutic effect by selectively binding to and inhibiting the MET receptor tyrosine kinase. By doing so, it blocks the receptor’s phosphorylation process initiated either by its ligand, hepatocyte growth factor, or by MET amplification. This inhibition reduces the downstream signaling (including pathways such as RAS/MAPK and PI3K/AKT) and ultimately impedes the growth and proliferation of MET-dependent cancer cells. Its ability to reach clinically meaningful plasma levels and demonstrate effective MET inhibition has been a cornerstone in its successful clinical development and eventual regulatory approvals.

Regulatory Approval Process

General Drug Approval Process Overview
The journey for a drug like Capmatinib begins with rigorous preclinical assessments followed by multiple phases of clinical trials (Phase 1 through Phase 3 and sometimes beyond). Regulatory agencies across different countries evaluate data pertaining to the drug’s pharmacodynamics, pharmacokinetics, safety, efficacy, and quality to determine if it should be approved for market use. In the case of Capmatinib, the clinical success demonstrated across international trials paved the way for regulatory submissions and approvals in major regions. For example, its first global approval was granted in the USA via the US Food and Drug Administration (FDA) in May 2020, establishing a precedent for subsequent submissions in other key markets.

Factors Influencing Drug Approval
Several factors influence the approval process for a drug like Capmatinib, including:
• Robust clinical trial data demonstrating significant efficacy (e.g., overall response rates of 68% in certain NSCLC populations) along with an acceptable safety profile, as seen in the GEOMETRY mono‑1 Phase II trial.
• The targeted nature of the treatment, especially in patients with unmet medical needs such as those with MET exon 14 skipping mutations, which is considered a driver mutation in NSCLC.
• Collaborative manufacturing and regulatory strategies by global subsidiaries (e.g., Novartis Pharmaceuticals Corp., Novartis Pharma KK, Novartis Europharm Ltd., and others) that tailor submissions to the specific requirements of each regulatory jurisdiction.
• Orphan drug or conditional approval designations in select regions, which are provided to facilitate the early availability of drugs addressing serious or life‑threatening conditions. For instance, Capmatinib received orphan drug status in both Australia and South Korea.

Capmatinib Approval Status by Country

Countries with Approval
Based on multiple high‑quality references from the synapse source and other structured clinical and regulatory documents, Capmatinib is approved in the following countries/regions:

• United States:
Capmatinib received its first global approval from the FDA in May 2020 for the treatment of adults with metastatic NSCLC whose tumors exhibit MET exon 14 skipping mutations. This initial approval set the stage for broader international marketing and was a critical milestone in establishing Capmatinib’s profile as a targeted therapy for this patient subset.

• European Union:
In Europe, Capmatinib’s development has been supported by applications submitted by Novartis Europharm Ltd. The drug application number “EMEA/H/C/004845” indicates that Capmatinib is approved in the EU through the centralized procedure managed by the European Medicines Agency (EMA), with an approval date listed as “2025-02-13.” While this may appear to be a future date relative to current timelines for some products, it reflects the planned or scheduled approval pathway in the European market. The comprehensive approval in the EU under the EMA guideline implies that Capmatinib will be marketed across all member states once the decision is finalized.

• Japan:
The Japanese approval comes via the Pharmaceutical and Medical Devices Agency (PMDA). The drug application record “30200AMX00494000” from Novartis Pharma KK confirms that Capmatinib (in tablet form under the trade names “TABRECTA tablets 150mg” and “タブレクタ錠150mg”) was approved on October 10, 2023. This approval highlights the localization of clinical development efforts to meet the specific standards and requirements of Japanese regulatory authorities.

• China:
Capmatinib has also been approved in China. The record with the drug application number “国药准字HJ20240041” indicates that the approval by the National Medical Products Administration (NMPA) was granted on June 11, 2024. Although the application organization is listed as Novartis Pharma Schweiz AG, this reflects the corporate strategy where regional subsidiaries support regulatory submissions. In China, Capmatinib is marketed under both the English trade name “Tabrecta” and a local name “妥瑞达,” ensuring its accessibility to the Chinese patient population.

• Australia:
In Australia, Capmatinib has achieved approval under an orphan drug designation for c‑Met positive non‑small cell lung cancer. This orphan drug approval was granted on January 11, 2022, reflecting the country’s emphasis on providing timely access to treatments that address rare or difficult‑to‑treat conditions. The orphan designation in Australia helps streamline the regulatory process, ensuring that patients with unmet needs can access the therapy sooner.

• South Korea:
Similarly, South Korea has approved Capmatinib as an orphan drug for the indication “MET Exon 14 Skipping Mutation Non‑small Cell Lung Cancer.” The approval date listed for South Korea is November 23, 2021. This development is significant as it confirms the commitment by South Korean regulatory bodies to evaluate and approve innovative treatments for patient subpopulations with specific genetic markers.

Pending Approvals
While Capmatinib is firmly approved in several major markets, there remain other potential markets where regulatory approval may be pending or under review. These may include other countries in Asia, additional constellations within the Middle East, or further regions that require additional local clinical data or specific manufacturing documentation that aligns with regional standards. In some instances, companies like Novartis often initiate rolling submissions or align subsequent approval strategies depending on local regulatory procedures, meaning that while the foundation is established in key markets (USA, EU, Japan, China, Australia, and South Korea), further expansion is anticipated as additional reviews conclude.

Implications of Approval

Market Impact
The approval of Capmatinib in these six major regions has several market implications. First, its status as a highly targeted therapy not only fills a critical gap in the treatment of MET exon 14 skipping NSCLC but also sets a precedent for similar precision medicines. The availability of Capmatinib in the USA, EU, Japan, China, Australia, and South Korea leads to increased competition among pharmaceutical companies, drives innovation in targeted therapy development, and contributes to global improvements in personalized cancer care. Moreover, the varied timelines—from its initial approval in the USA in 2020 to later approvals in Japan (2023) and China (2024)—demonstrate both the challenges and the coordinated efforts across different regulatory environments. These successes have triggered downstream benefits such as milestone payments to Incyte and broader licensing opportunities, further bolstering the market position of Capmatinib.

Accessibility and Treatment Options
From a patient-access perspective, the approval in multiple countries means that patients across different regions can access an effective treatment option. This is particularly important in diseases like NSCLC with MET exon 14 skipping mutations, where traditional treatment options were limited. In the USA, the approval by the FDA has already translated into expanded treatment options for patients with advanced NSCLC. In the EU, once the EMA decision is fully executed, similar accessibility will be achieved across member states. Additionally, the orphan designations in Australia and South Korea help reduce the regulatory burden and speed up access to these novel therapies for patients with rare genetic variations, ultimately leading to improved outcomes and quality of life. The approval in China, one of the largest pharmaceutical markets, is particularly impactful as it ensures that a substantial patient base can receive personalized treatment tailored to their genetic profile. Finally, the Japanese market approval signifies strong regional confidence in Capmatinib’s efficacy and safety profile, further supporting its global roll‑out strategy.

In broader terms, the hierarchical expansion of regulatory approvals across these diverse markets not only increases the global availability of Capmatinib but also encourages continued clinical development and post‑marketing research to refine treatment regimens and monitor long‑term patient outcomes. This multimarket approval strategy paves the way for subsequent collaborations, further clinical trials in untargeted indications, and potentially expansion into additional markets where approval may be forthcoming.

Conclusion
In summary, Capmatinib has successfully traversed the complex landscape of global regulatory approval and is now approved in several key regions: the United States (FDA, May 2020), the European Union (via EMA with a planned approval date as indicated in the application record), Japan (PMDA approval on October 10, 2023), China (NMPA approval on June 11, 2024), Australia (orphan drug approval on January 11, 2022), and South Korea (orphan drug approval on November 23, 2021). This diverse regulatory landscape reflects not only the broad clinical utility of Capmatinib in treating MET exon 14 skipping NSCLC but also the strategic efforts by Novartis to meet the varying requirements of global regulatory bodies. The implications of these approvals are significant: they signal improved accessibility to targeted cancer therapies, stimulate competition and innovation in the oncology market, and ultimately enhance treatment outcomes for patients with challenging and rare oncogenic drivers.

Going forward, while approvals in several major markets have been secured, additional regulatory submissions may be pending in other regions, further expanding the reach of Capmatinib. With its robust clinical efficacy and safety data supported by extensive global trials, Capmatinib stands at the forefront of precision oncology, offering renewed hope for patients worldwide and setting a benchmark for future targeted therapies.