Bridging the Pediatric Drug Gap: Pudong Pharma Firms Lead Innovation and Collaboration

In recent years, China has accelerated a wave of policies aimed at addressing a critical gap in the pharmaceutical industry: pediatric medications. On May 7, eight government agencies including the National Health Commission jointly issued implementation guidelines to reform and improve the supply mechanism for children's medicines. Just eight days later, the newly revised Drug Administration Law Implementation Regulations took effect, establishing for the first time a market exclusivity period system specifically for pediatric drugs—an administrative measure designed to incentivize research and development.

The National Medical Products Administration has also launched a special pilot program called the Starlight Plan, targeting the persistent challenges of developing pediatric oncology drugs—namely, the difficulties in research and the slow pace of market approval.

These policy tailwinds have sparked a series of breakthroughs in pediatric drug development in Pudong, Shanghai's innovation hub. Leading the charge is Fosun Pharmaceutical, whose self-developed MEK 1/2 inhibitor lumezemetinib tablets (brand name: Fumaining®) received approval from China's National Medical Products Administration on June 15. The drug is indicated for pediatric and adolescent patients aged 2 years and older with relapsed or refractory Langerhans cell histiocytosis (LCH) following systemic treatment—making it the only approved therapy for this indication in China.

Langerhans cell histiocytosis is an inflammatory neoplastic disease caused by the clonal expansion of CD207-positive histiocytes of myeloid origin. The World Health Organization classifies it as a hematopoietic system tumor, and it represents the most common histiocytic disorder in children. Traditional chemotherapy has shown limited efficacy with significant toxic side effects, leaving approximately 30% to 40% of surviving children with lifelong disabilities. The approval of Fumaining® offers a new targeted therapy option for this rare but deadly condition.

In September 2025, this pediatric LCH indication, along with low-grade glioma, was included in the Starlight Plan pilot project for pediatric oncology drugs by the Center for Drug Evaluation.

Meanwhile, thederma has achieved a significant milestone in treating infant atopic dermatitis. The company's self-developed benvitimod cream (brand name: Zelimei®) became the first globally approved aromatic hydrocarbon receptor (AhR) modulator non-hormonal topical medication for pediatric atopic dermatitis. The product received approval in November 2024.

Atopic dermatitis, commonly known as eczema, is the most prevalent dermatological condition. In China, the prevalence among children aged 1 to 7 years reaches 12.94%, while among infants under 1 year old, the rate climbs to 30%—meaning roughly one in three babies may suffer from the condition.

thederma took on the challenge of addressing this unmet clinical need. The company successfully completed the world's first Phase III clinical trial in children aged 3 months to 2 years—a low-age group that had previously been excluded from such studies. The results were striking: 81.6% of pediatric patients achieved 90% or greater improvement or clearance of skin lesions (EASI 90).

"This is a breakthrough achievement," said Professor Zhang Jianzhong from the Department of Dermatology at Peking University People's Hospital. "This Phase III clinical study demonstrates that for pediatric atopic dermatitis, topical treatment is the fundamental therapy. Benvitimod represents a small step, but China has taken a giant leap forward. Through new drug development, we are addressing the long-standing inadequacies in atopic dermatitis treatment and even leading international topical therapy. This research aligns with national policy directives for pediatric drug innovation and injects strong momentum into China's children's health and domestic pharmaceutical industry innovation."

Beyond these headline achievements, Pudong has emerged as a diversified ecosystem for pediatric drug development, with companies tackling conditions ranging from respiratory syncytial virus (RSV) infections to growth hormone deficiencies.

ArkBio, founded in Zhangjiang in 2014, has focused on addressing major unmet clinical needs in respiratory and pediatric diseases. In early 2026, the company's ADHD medication Aizhida® received approval for patients aged 6 years and older with attention deficit hyperactivity disorder. It is the world's first and only combination formulation containing both immediate-release dexmethylphenidate (d-MPH) and the prodrug serdexmethylphenidate (SDX). Each capsule of the compound dexsermethylphenidate contains 70% SDX and 30% d-MPH.

ArkBio's other core product, qirisovir—a new RSV antiviral—has been submitted for market approval to the National Medical Products Administration and has been included in the World Health Organization's priority list for optimized pediatric RSV medications.

VISEN Pharmaceuticals has made strides in pediatric endocrinology. Its core product, Weizhengao® (lonapegsomatropin injection), received approval in January 2026 for the treatment of growth failure in children and adolescents aged 3 years and older caused by growth hormone deficiency. The product utilizes TransCon proprietary technology to enable once-weekly dosing.

In a head-to-head Phase III clinical trial known as the briGHt study, VISEN's product achieved an annualized growth rate of 10.66 centimeters per year, significantly outperforming the control group's 9.75 centimeters per year. It is currently the only long-acting growth hormone that has demonstrated statistically significant superiority over daily formulations in two pivotal Phase III clinical trials. The results of the briGHt study were published in the international core journal Hormone Research in Paediatrics.

Aokeda Pharma has developed a proprietary AUCped (pediatric formulation) technology platform dedicated to creating improved new medications better suited for children, with the goal of enhancing medication compliance and tolerance. The company's AUC033 (Motopoly XR) is the world's first and only once-daily extended-release lacosamide (ER capsule) approved by the U.S. Food and Drug Administration. Given that epilepsy treatment requires long-term, regular medication, extended-release formulations are critical for addressing the clinical challenges of traditional immediate-release formulations, including significant fluctuations in blood drug concentration and poor patient compliance.

Beyond drug innovation, Pudong is accelerating the integration of industry, academia, research, and clinical practice to create a closed-loop ecosystem of modular resonance and medical-engineering synergy.

Pediatric disease-specific cohort research data represents a core strategic resource for supporting disease mechanism research, target discovery, biomarker screening, and innovative therapy development. It also serves as a critical driver for advancing basic scientific research and clinical translation.

Recently, the National Center for Children's Health (Shanghai Children's Medical Center affiliated with Shanghai Jiao Tong University School of Medicine), the Shanghai Data Exchange, Aopu Shuchuang Biology, and GeneScience Pharmaceuticals officially signed an agreement to jointly build a leading domestic pediatric disease-specific cohort research data product system. The collaboration will explore a dual-track compliant circulation model combining "technology services and data products."

With the partnership established, the parties will accelerate project implementation and are expected to produce the first batch of shareable research data results within the year. This model will equip pediatric basic research and clinical translation with a "data engine," provide high-quality compliant data for biopharmaceutical companies to accelerate innovative therapy development for rare pediatric diseases and tumors, and—through demonstration practices—offer a replicable and promotable Pudong sample and Shanghai solution for the market-oriented reform of medical health data elements nationwide.

Research indicates that globally, Asia and Oceania bear the heaviest pediatric tumor burden, with China ranking second in the region. Among all tumor-related disease burdens worldwide, pediatric tumors rank sixth, following only adult lung cancer, liver cancer, gastric cancer, colon cancer, and breast cancer.

As Pudong continues to break new ground in pediatric medication, the district is not just filling gaps in treatment—it is positioning itself as a model for how targeted policy, innovative research, and collaborative ecosystems can address one of medicine's most challenging frontiers.