Alecensa Receives the World-First Tumor-Agnostic Approval for ALK Fusion Gene-Positive Solid Tumors Across Adult and Pediatric Patients

• The world’s first tumor-agnostic therapy covering pediatric indications for ALK fusion gene-positive solid tumors
• Approval based on results from an investigator-initiated Phase II clinical study in Japan evaluating the efficacy and safety of Alecensa in patients with rare cancers with advanced or recurrent ALK gene alterations

TOKYO, May 18, 2026 -- Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) today announced that it received regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for the additional indication of its anti-cancer agent/ALK inhibitor Alecensa^® (generic name: alectinib) for advanced or recurrent ALK fusion gene-positive solid tumors, including pediatric patients. This is the world’s first tumor-agnostic approval for an ALK inhibitor.

“We are delighted that Alecensa, discovered by Chugai, has been approved as the world’s first tumor-agnostic therapy for patients with ALK fusion-positive solid tumors.
This approval expands access to Alecensa beyond its established use in non-small cell lung cancer and anaplastic large cell lymphoma, bringing a new treatment option to patients across a wide range of cancer types. We will continue striving to further contribute to personalized healthcare, delivering the most appropriate treatment to each patient, regardless of cancer type or age,” said Chugai’s President and CEO, Dr. Osamu Okuda.

This approval is based on the results of the TACKLE study, an investigator initiated Japanese Phase II clinical study evaluating the efficacy and safety of Alecensa in pediatric and adult patients with rare cancers harboring ALK gene abnormalities (fusion / rearrangement genes, activating mutations, and gene copy number amplification) in advanced or recurrent settings. In this study, the response rate as assessed by the central review committee, which was the primary endpoint, was 43.8% (7/16 patients, 95% CI: 19.8%-70.1%) in the full analysis set (FAS) of the main cohort. In the ALK fusion gene-positive subpopulation within this cohort, the response rate was 70.0% (7/10 patients, 95% CI: 34.8%–93.3%). In addition, in the ALK fusion gene-positive subpopulation of the overall FAS pooled across all cohorts, the response rate was 76.5% (13/17 patients, 95% CI: 50.1%-93.2%), and the efficacy data in patients with ALK fusion gene-positive tumors from this study were considered the key basis for regulatory approval. The incidence of adverse events was 73.1% (19/26 patients), with the main adverse reactions being lymphocyte count decreased, and neutrophil count decreased at 23.1% each (6/26 patients), anemia at 19.2% (5/26 patients), and blood creatinine increased at 15.4% (4/26 patients). The safety profile observed in this study was similar to the previously established safety profile of Alecensa, with no new safety signals identified.

FoundationOne^® CDx Cancer Genomic Profile is used as a companion diagnostic to identify people that could potentially benefit from Alecensa for ALK fusion-positive solid tumors. The MHLW granted the approval for the expanded use of FoundationOne^® CDx Cancer Genomic Profile as a companion diagnostic of Alecensa on March 9, 2026.

Chugai Pharmaceutical, a leading company in the oncology field, remains committed to addressing unmet medical needs in cancer treatment with innovative medicines for patients and healthcare professionals.

Approval Information *Relevant sections only, with modifications underlined

Indications:
◯ ALK fusion-positive advanced or recurrent solid tumors
◯ Adjuvant treatment in ALK fusion-positive non-small cell lung cancer
◯ ALK fusion-positive recurrent or refractory anaplastic large cell lymphoma

Dosage and Administration:
<ALK fusion-positive advanced or recurrent solid tumors>
The usual adult dosage is 300 mg alectinib administered orally twice a day.
The usual pediatric dosage of alectinib is based on body weight as shown below and administered orally once or twice daily.

[Reference Information]

Chugai Files for Additional Tumor-Agnostic Indication of Alecensa for ALK Fusion / Rearrangement Gene-Positive Solid Tumors Including Pediatric Patients (News release issued on June 26, 2025)
https://www.chugai-pharm.co.jp/english/news/detail/20250626153000_1167.html

Chugai Obtains Approval for FoundationOne CDx Cancer Genomic Profile as a Companion Diagnostic of Alecensa for ALK Fusion Gene-Positive Solid Tumors (News release issued on May 18, 2026)
https://www.chugai-pharm.co.jp/english/news/detail/20260518153000_1244.html

About the TACKLE study

The TACKLE study (NCCH1712/MK003, jRCT2091220364) is an investigator initiated, multicenter, open-label, single-arm, Japanese Phase II clinical study evaluating the efficacy and safety of Alecensa in pediatric and adult patients with unresectable rare cancers harboring ALK gene abnormalities (fusion / rearrangement genes, activating mutations, gene copy number amplification). In this study, safety and efficacy were evaluated in 26 patients, utilizing genomic information, including data registered with the Center for Cancer Genomics and Advanced Therapeutics (C-CAT). The primary endpoint was response rate, and secondary endpoints included progression-free survival, overall survival, and safety. The TACKLE study is being conducted as a substudy of the MASTER KEY project,¹ which promotes the development of treatments for rare cancers through industry-academia collaboration led by the National Cancer Center Hospital, and is being carried out at four sites in Japan: National Cancer Center Hospital, Kyoto University Hospital, Kyushu University Hospital, and Hokkaido University Hospital.

About ALK fusion / rearrangement gene-positive solid tumors

ALK fusion / rearrangement genes are abnormal genes created when the ALK (anaplastic lymphoma kinase) gene fuses with other genes (such as EML4, NPM) as a result of chromosomal translocation.^2,3 ALK fusion / rearrangement proteins produced from these fusion / rearrangement genes are thought to promote cancer cell proliferation. ALK fusion / rearrangement genes have been identified in patients with inflammatory myofibroblastic tumors, lung cancer, breast cancer, colorectal cancer, and other cancers.^2,4,5

About Alecensa

Alecensa is an oral medicine discovered by Chugai, which is highly selective for ALK and active in the central nervous system. ALK fusion / rearrangement gene-positive lung cancer is found in approximately 3-5% of NSCLC cases.⁴ Alecensa is already approved in over 100 countries as an initial (first-line) and second-line treatment for ALK fusion / rearrangement gene-positive metastatic NSCLC, including in the United States, Europe, Japan, China, and Taiwan. For adjuvant therapy of ALK fusion / rearrangement gene-positive NSCLC, Alecensa received approval in the United States in April 2024, followed by Europe in June 2024, and Japan in August 2024. In Japan, Alecensa has also been approved for the treatment of recurrent or refractory ALK fusion / rearrangement gene-positive anaplastic large cell lymphoma.

Trademarks used or mentioned in this release are protected by law.

Sources:

1. National Cancer Center Hospital in Japan. MASTER KEY Project Overview [Internet; cited May 2026]. Available from: https://www.ncc.go.jp/jp/ncch/masterkeyproject/outline/overview/master_key_project_overview.html.
2. Morris, SW. et al.: Fusion of a kinase gene, ALK, to a nucleolar protein gene, NPM, in non-Hodgkin’s lymphoma. Science 267(5196): 316-317, 1995.
3. Soda, M. et al.: Identification of the transforming EML4-ALK fusion gene in non-small-cell lung cancer. Nature 448(7153): 561-566, 2007.
4. The Japan Lung Cancer Society. Guide to various lung cancer biomarker tests 4) Genes targeted by biomarker testing and their abnormalities 4-2. ALK (Published 2.0 of April 2025) (Japanese only) [Internet; cited May 2026] Available from:
   https://www.haigan.gr.jp/publication/guidance/inspection/
5. Lin, E. et al.: Exon array profiling detects EML4-ALK fusion in breast, colorectal, and non-small cell lung cancers. Mol. Cancer Res. 7: 1466-1476, 2009.