Shanghai, China, today —Haihe Biopharma Co., Ltd. (“HaiHe”) announced that its highly selective PI3Kα inhibitor (development code: CYH33) has been granted orphan drug designation by Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of advanced or recurrent ovarian clear cell carcinoma (OCCC) harboring PIK3CA gene mutations that has progressed after chemotherapy.
Dr. Ruiping Dong, Chief Executive Officer of Haihe Biopharma, stated,
We are glad that CYH33 has received orphan drug designation from Japan’s MHLW, marking another milestone in Haihe’s global pursuit of precision medicine. Patients with ovarian clear cell carcinoma (OCCC) have limited treatment options, highlighting a substantial unmet medical need. As the world’s first monotherapy targeting PIK3CA-mutated OCCC, CYH33 is expected to provide a novel treatment option. This orphan drug designation will accelerate the regulatory approval process for the drug's Japanese market entry. We will continue to strive for CYH33's approval and commercialization. Moving forward, Haihe remains committed to addressing unmet medical needs and advancing China-originated innovative therapies to benefit patients worldwide, upholding our mission of 'patient-centric innovation'.
Dr. Xiaohua Wu, the leading principal investigator of CYH33-G201 from Fudan University Shanghai Cancer Center, commented,
As a rare subtype of cancer, OCCC exhibits more aggressive biological behavior than the most common serous ovarian cancer. It is characterized by a unique molecular profile and clinical features, with the majority of cases exhibiting resistance to platinum-based chemotherapy and a poor prognosis, representing an unmet medical challenge. CYH33, the first selective PI3Kα inhibitor developed for OCCC, has shown potent anti-tumor efficacy in clinical studies, providing a new treatment option for OCCC patients. This orphan drug designation is a recognition of its clinical value and marks a breakthrough for China’s innovative drugs on the global stage. We look forward to CYH33 reshaping the treatment landscape of OCCC and hope that China's health authorities will prioritize the R&D of innovative drugs for rare malignant tumors like OCCC.
About Orphan Drug Designation in Japan
Under the provisions of the Pharmaceuticals and Medical Devices Act, drugs designated as orphan drugs by the Japanese Ministry of Health, Labour and Welfare (MHLW) are entitled to multiple policy supports, including expedited review. The designation must meet the following criteria:
1.The number of patients affected domestically must be fewer than 50,000 (4/100,000).
2.The drug is intended for treatment of a serious disease that lacks alternative therapies or, compared to existing drugs, is expected to demonstrate significantly improved efficacy and safety, addressing a high unmet medical need.
3.The application of the drug for treating the target disease must be based on theoretically sound scientific grounds, while its development plan must be confirmed as scientifically justified and feasible.
About ovarian clear cell carcinoma and PIK3CA mutations
Ovarian clear cell carcinoma (OCCC) is a rare and highly aggressive subtype of epithelial ovarian cancer. Compared to the more common serous ovarian carcinoma, OCCC exhibit distinct molecular features and have significantly lower response rates to platinum-based chemotherapy. For recurrent OCCC, no standardized treatment regimen exists, and its prognosis is markedly worse than that of serous ovarian carcinoma, highlighting a significant unmet clinical need.
Genomic studies have revealed that PIK3CA mutations are highly prevalent in OCCC. These mutations are predominantly enriched in exon 9 (helical domain) and exon 20 (kinase domain), leading to aberrant activation of PI3Kα and its downstream signaling pathways, which drive OCCC tumorigenesis and progression. As such, PI3Kα represents a promising therapeutic target for OCCC.
About Risovalisib (CYH33)
CYH33 is a highly selective phosphatidylinositol 3-kinase α (PI3Kα) inhibitor with a novel structure independently developed by Haihe Biopharma with global intellectual property rights. Preclinical studies demonstrated that CYH33 can effectively and selectively inhibit PI3Kα kinase activity, and have exhibited potent anti-tumor activity in multiple PIK3CA-mutated tumor models. Preliminary data from CYH33 clinical trials suggested that CYH33 has a manageable safety profile and encouraging efficacy in treating a variety of advanced solid tumors with PIK3CA mutations, including breast cancer, ovarian cancer, and endometrial cancer. Currently, multiple clinical studies of CYH33 either as a single agent or in combination with other antitumor agents are ongoing to evaluate its safety and efficacy across a broader spectrum of advanced cancer patients.
About Haihe Biopharma Co., Ltd.
Haihe Biopharma Co., Ltd. is a global, innovation-driven biopharmaceutical company headquartered in China, with operational hubs in the United States and Japan. The company is dedicated to the development of cutting-edge anti-cancer therapies. With fully integrated capabilities spanning drug discovery, development, manufacturing, and commercialization, Haihe Biopharma aims to deliver life-saving treatments to cancer patients around the world and features a globally minded management and R&D team focused on in-house innovation. Currently, Haihe Biopharma has two approved products: Gumarontinib (INN), approved in both China and Japan, and paclitaxel oral solution (RMX3001/DHP107), approved in China, along with a robust pipeline of additional drug candidates.
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