Otsuka Pharmaceutical Co., Ltd. (Otsuka) announceｓ that it has submitted a supplemental application in Japan seeking regulatory approval for an expanded indication of Iclusig^® (ponatinib hydrochloride) to include first-line treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in adult patients. In conjunction with this application, Otsuka has also submitted a formulation addition for Iclusig 10 mg tablets, which will be required for use in first-line treatment.

Iclusig is an oral tyrosine kinase inhibitor originally discovered by Ariad Pharmaceuticals, Inc., which was acquired by Takeda in 2017. Otsuka holds the rights for the co-development and commercialization of Iclusig across nine Asian countries and regions, including Japan.

The product was approved in Japan in 2016 for the treatment of chronic myeloid leukemia (CML) refractory or intolerant to prior therapies, and relapsed or refractory Ph+ ALL.

Ph+ ALL is an aggressive form of leukemia characterized by the presence of the Philadelphia chromosome, which produces the BCR::ABL1 tyrosine kinase, a key driver of disease progression. Leukemic blasts--malignant immature lymphoid cells--proliferate primarily in the bone marrow, leading to rapid clinical deterioration. In the United States, Iclusig (U.S. product name: Iclusig) initially received approval for use as monotherapy in adult Ph+ ALL patients who are T315I-positive or for whom no other tyrosine kinase inhibitor (TKI) is indicated. In 2024, the U.S. FDA granted accelerated approval for Iclusig in combination with chemotherapy for first-line treatment of Ph+ ALL, further expanding its clinical utility.

In Japan, treatment options for first-line management of Ph+ ALL remain limited, and there is a significant need for additional therapies to improve clinical outcomes. The newly submitted indication is therefore expected to provide an important new treatment option for patients with this high-risk leukemia.

Otsuka remains committed to addressing unmet medical needs by delivering innovative therapeutic options and contributing to improved outcomes for patients with serious diseases.

About Iclusig

Iclusig is a tyrosine kinase inhibitor that targets the abnormal BCR::ABL1 tyrosine kinase expressed in CML and Ph+ ALL. It inhibits both wild-type BCR::ABL1 and clinically-relevant single mutations that confer resistance to other therapies, including the T315I mutation, and demonstrates efficacy in CML and Ph+ ALL.

Iclusig received initial approvals in the United States in 2012 and in Europe in 2013. Otsuka obtained approval in Japan in 2016 and began marketing in the same year. The drug was designated as an orphan drug in Japan for CML resistant or intolerant to prior drug treatment and relapsed or refractory Ph+ ALL in 2015 and received additional orphan designation for Ph+ ALL in 2026. The first-line indication for adult patients with Ph+ ALL in combination with chemotherapy received accelerated approval in the U.S. in 2024, followed by approvals in several other countries, including those in Asia.

About Otsuka

Otsuka Pharmaceutical Co., Ltd. is a total healthcare company that focuses on each individual's potential to enhance their well-being. Our medical-related business provides treatments and diagnostics for both physical and mental health. Our nutraceutical business supports daily health maintenance and improvement. Otsuka's unique products and services are based on scientific evidence, under the guidance of our corporate philosophy: Otsuka-people creating new products for better health worldwide. For further information, please visit www.otsuka.co.jp/en/.