Singapore and Princeton, New Jersey, U.S., November 11, 2026
Shanton Pharma has announced that it has secured important regulatory agreements with the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) concerning pediatric clinical study requirements for SAP-001, its investigational oral treatment for hyperuricemia and gout. The EMA has agreed to a product-specific full waiver of pediatric investigation requirements across pediatric age groups for hyperuricemia of causes other than chronic kidney disease or obesity. This follows the FDA’s agreement on the initial Pediatric Study Plan submitted in May 2026, which exempts SAP-001 from pediatric study requirements based on the extremely low prevalence of gout in children. The regulatory decisions represent an important development milestone for the clinical-stage biotechnology company as it advances its lead drug candidate toward further clinical development and potential regulatory submissions. By removing the need for certain pediatric clinical studies, the waivers are expected to reduce development costs, administrative requirements and regulatory complexity while allowing the company to concentrate its resources on evaluating SAP-001 in adult patients with uncontrolled or refractory gout.
EMA and FDA Grant Pediatric Study Waivers
The latest regulatory announcement confirms that both the EMA and FDA have agreed that pediatric clinical studies of SAP-001 are not required to support future marketing authorization applications for the relevant hyperuricemia and gout indications. Under the EMA agreement, Shanton received a product-specific full waiver covering pediatric age subsets with hyperuricemia of causes other than chronic kidney disease or obesity. This decision forms part of the company’s agreed Pediatric Investigation Plan (PIP), which establishes the regulatory approach to pediatric medicine development in Europe. The FDA decision follows the company’s submission of an initial Pediatric Study Plan in May 2026. The agency agreed to exempt SAP-001 from pediatric study requirements because gout is extremely uncommon in children. These decisions are significant because they help avoid unnecessary pediatric clinical investigations when the expected medical need and disease prevalence do not justify such studies. The regulatory waivers are expected to streamline SAP-001’s development programme, reducing the financial and operational burden associated with additional pediatric research. However, these decisions do not represent marketing approval or an exemption from clinical development and regulatory requirements for the intended adult population.
SAP-001 Targets Uncontrolled Gout and Hyperuricemia
SAP-001 is an investigational once-daily oral urate-lowering therapy being developed to address unmet medical needs among adults with uncontrolled or refractory gout. The drug candidate is based on a novel mechanism of action intended to reduce elevated uric acid levels in patients who do not respond adequately to conventional urate-lowering treatments or cannot tolerate available therapies. Hyperuricemia occurs when excessive uric acid accumulates in the blood. Elevated uric acid can lead to the formation of monosodium urate crystals in joints and surrounding tissues, resulting in gout attacks characterized by severe pain, swelling, redness and inflammation. Repeated attacks can contribute to chronic joint damage and the development of tophi, which are deposits of urate crystals under the skin and around joints. Existing treatment approaches commonly include xanthine oxidase inhibitors, which reduce uric acid production. However, some patients continue to experience uncontrolled uric acid levels despite standard treatment. This creates a significant need for alternative therapeutic approaches. Shanton is developing SAP-001 as a potential first-in-class oral treatment for patients with refractory gout. According to the company, the drug candidate demonstrated encouraging efficacy and safety findings in a Phase 2b clinical study involving patients with hyperuricemia who had inadequate responses to standard-of-care xanthine oxidase inhibitor therapy.
Advertisement
Phase 2b Results Support Further Clinical Development
The development programme for SAP-001 has achieved several regulatory and clinical milestones. In July 2025, the investigational medicine received FDA Fast Track designation for the treatment of hyperuricemia in adults with gout who are refractory to conventional therapy. The designation was supported in part by Phase 2b findings demonstrating the potential of SAP-001 to improve treatment outcomes in patients with uncontrolled gout. In October 2025, Shanton also announced the successful completion of an End-of-Phase 2 meeting with the FDA. The meeting provided alignment on important aspects of the proposed Phase 3 programme, including clinical trial design, patient population, dose selection, endpoints and statistical analysis methods. This regulatory interaction helped establish a pathway toward the company’s planned multinational clinical development programme. Bing Li, Ph.D., Chief Executive Officer of Shanton Pharma, stated that the agreements with the EMA and FDA represent an important achievement for the company’s SAP-001 programme. The decisions are expected to reduce unnecessary clinical development requirements and support more efficient advancement of the investigational treatment. Shanton Pharma is a clinical-stage biotechnology company founded in 2016, with its headquarters in Singapore and research and development activities across the United States, China and Singapore. The latest regulatory milestone strengthens the company’s development strategy for SAP-001 and highlights the importance of regulatory collaboration in pharmaceutical research. Although the pediatric waivers simplify certain development requirements, further clinical evaluation and regulatory review will remain necessary before SAP-001 can become an approved treatment for adults with refractory gout. If future studies confirm its safety and effectiveness, the investigational medicine could provide an additional treatment option for patients who continue to experience uncontrolled hyperuricemia despite existing therapies.
Source: Shanton Pharma press release
