Two Drugs Enter Japan, Haihe Pharmaceutical Advances Early Entry into Pediatric Drug Market

Ask AI · What opportunities are there in the deep water of pediatric drug development?

The 2026 National Two Sessions have concluded successfully. This year, biopharmaceuticals were included in the “Emerging Pillar Industries” for the first time and incorporated into the government work report, establishing an unprecedented strategic position.

Data shows that by 2025, China had approved a total of 76 new drugs, with domestic companies licensing out products worth a total of $130 billion, making China an important source of global pharmaceutical innovation. In the first two months of 2026, the total value of licensing transactions for innovative drugs exceeded $50 billion, more than one-third of the total for 2025, indicating a strong momentum for overseas expansion.

The leap in China’s pharmaceutical innovation capability is attributed not only to sustained government support for the biopharmaceutical industry but also to the proactive efforts of domestic companies to expand globally and deepen innovation R&D. As early as 2024, when most Chinese pharmaceutical companies were still exploring overseas markets, innovative drug companies like Haihe Pharmaceuticals had already begun to make a mark on the international stage. As a company focused on oncology, Haihe Pharmaceuticals has developed differentiated competitiveness in niche areas and is one of the leaders in the domestic small-molecule innovative drug sector. Under the guidance of favorable policies this year, Haihe Pharmaceuticals has achieved new breakthroughs.

Japan is the third-largest pharmaceutical market in the world, known for its strict and cautious drug regulatory system. Gaining approval in Japan is an important validation of a pharmaceutical company’s R&D strength and international compliance capabilities. Haihe Pharmaceuticals is the first domestic innovative biotech company to successfully open the Japanese market, and with solid independent innovation and global development capabilities, it has continuously launched products.

In June 2024, Haihe’s self-developed MET inhibitor, Gumeitene tablets, was launched in Japan, becoming the first Chinese innovative drug developed and approved by a Chinese company to enter the Japanese market. On March 23, 2026, its self-developed PI3Kα selective inhibitor, Risoletin mesylate tablets, was approved by Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of ovarian clear cell carcinoma (OCCC) with PIK3CA gene mutations after chemotherapy. Risoletin is the world’s first single-agent targeted therapy for post-chemotherapy progression in OCCC and the first PI3Kα inhibitor approved in Japan.

With two innovative drugs successfully launched overseas in two years, Haihe Pharmaceuticals’ continuous innovation fully demonstrates its strength as a benchmark enterprise in China’s small-molecule innovation drugs and its determination to go global.

Risoletin Expands Imagination

Ovarian clear cell carcinoma (OCCC) is a rare and highly aggressive epithelial ovarian cancer subtype, accounting for about 5%–25% of all ovarian cancers, more common among Asians. Compared to the more common serous ovarian cancer, OCCC has unique molecular features, limited efficacy with traditional treatments, significantly lower response rates to platinum-based chemotherapy, and no standard treatment options upon recurrence. Genomic studies show that high-frequency PIK3CA mutations are a key driver of tumor development. Risoletin targets this mutation and achieved a breakthrough early on. In June 2025, Risoletin received orphan drug designation from Japan’s Ministry of Health, Labour and Welfare, and on March 23, it was approved for marketing in Japan, filling the clinical treatment gap for PIK3CA-mutated ovarian clear cell carcinoma. Professor Wu Xiaohua from Fudan University Affiliated Tumor Hospital, a global principal investigator for Risoletin, stated: “This approval not only affirms the drug’s clinical value but also redefines the treatment landscape for OCCC with Chinese innovation, highlighting the strength of domestic innovative drugs globally.” Haihe Pharmaceuticals had already completed its commercialization layout in Japan early. Last October, the company reached an exclusive licensing agreement for Risoletin’s development, production, and commercialization in Japan. Previously, Gumeitene’s commercialization in Japan also followed this model, enabling rapid transition from approval to market entry.

Beyond OCCC, Haihe Pharmaceuticals is also expanding internationally into other unmet clinical areas such as PIK3CA-related overgrowth spectrum (PROS) and PIK3CA-related vascular malformations (PRVM).

Venture into the Deep Water of Pediatric Drug Development

PIK3CA gene somatic mutations can cause a series of rare and severe overgrowth diseases. Among them, PROS (PIK3CA-related overgrowth spectrum) patients often exhibit unilateral body or limb (hand, foot, leg) hypertrophy; some experience severe symptoms like epilepsy and intellectual disability due to brain overgrowth. PRVM (PIK3CA-related vascular malformations) mainly features abnormal development of blood and lymphatic vessels, leading to complex vascular malformations. Both PROS and PRVM are mostly found in children, with PROS included in the National Health Commission’s “Second Batch of Rare Disease List.” Patients have long lacked effective systemic treatments and can only rely on repeated surgeries to alleviate symptoms, suffering significant physical and mental pain.

Haihe Pharmaceuticals was among the early companies to recognize this high unmet clinical need and is one of the few global pharmaceutical companies actively developing in this area. Currently, Risoletin is in Phase II pivotal clinical trials for PROS/PRVM, being conducted simultaneously in China and Japan.

Pediatric drug development is traditionally a “deep water” area. Challenges include small patient populations, high clinical trial thresholds, strict ethical requirements, and far greater difficulty compared to adult drugs. Few innovative companies have a forward-looking layout in this field. Haihe Pharmaceuticals’ efforts in pediatric rare diseases have also received strong policy support. In May 2025, the National Medical Products Administration (NMPA) launched the “Encouragement Pilot Program for Pediatric Oncology Drug Development (Starlight Program),” and Risoletin was invited to join due to its unique therapeutic value, potentially accelerating clinical development and approval, and injecting key momentum into filling the treatment gap for pediatric rare diseases.

Risoletin Selected for the “Starlight Program”

Source: National Medical Products Administration official website

In January 2026, the new “Regulations on the Implementation of the Drug Administration Law” also officially took effect, stipulating that pediatric drugs can enjoy up to 2 years of market exclusivity, and rare disease drugs up to 7 years.

During the 2026 National Two Sessions, deputies and members also made suggestions for ensuring pediatric and rare disease drug access. Dingle Ming, a member of the Chinese People’s Political Consultative Conference, called for faster establishment of “special channels” for rare disease and pediatric drugs, promoting “market approval upon listing,” and timely inclusion in medical insurance at appropriate prices to benefit patients early.

With strong policy support, Haihe Pharmaceuticals’ path in pediatric and rare disease drug development is no longer lonely and is becoming increasingly motivated.

Conclusion:

Chinese innovative drugs are shifting from “following” to “running alongside,” and even beginning to “lead” in certain niche areas. From domestic approvals to opening the Japanese market twice, from filling domestic gaps to participating in global pricing, every step taken by Haihe Pharmaceuticals proves that China’s pharmaceutical companies’ competitiveness has shifted from cost advantages to innovation advantages.

As more companies like “Haihe” step onto the global stage, the next chapter of Chinese innovative medicine truly begins.

Written by: Li Ao

Edited by: Jiang Yun, Jia Ting

Operations: Li Muzi

Note: Original content by Jian Shi Ju. Please do not reproduce without permission.