GALASHIELS & MARLOW, United Kingdom, July 21, 2026
Kyowa Kirin EMEA has announced that the European Commission (EC) has approved an expanded indication for Crysvita® (burosumab), allowing its use in infants aged one month to one year with X-linked hypophosphataemia (XLH) across the European Union (EU) and European Economic Area (EEA). The regulatory milestone marks a significant advancement in the treatment of XLH, a rare, progressive genetic disorder that disrupts phosphate metabolism and impairs bone mineralization from infancy. By enabling treatment at an earlier stage of life, the approval provides physicians with the opportunity to intervene before irreversible skeletal complications develop, supporting improved long-term growth and physical function. The expanded indication strengthens Crysvita’s position as the first targeted therapy approved for this vulnerable patient population while reinforcing Kyowa Kirin’s commitment to advancing innovative therapies for rare diseases. The approval follows a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and is supported by clinical evidence demonstrating the therapy’s safety and efficacy in infants.
Earlier Crysvita Treatment Expands Care for Infants with XLH
The expanded approval allows healthcare professionals throughout Europe to initiate Crysvita (burosumab) treatment from as early as one month of age, addressing a critical unmet medical need in infants diagnosed with X-linked hypophosphataemia. XLH is caused by excessive activity of fibroblast growth factor 23 (FGF23), resulting in chronic phosphate wasting, impaired bone mineralization, skeletal deformities, delayed growth, and long-term physical disability if left untreated. Since disease manifestations often begin during infancy, earlier therapeutic intervention is considered essential for preserving normal skeletal development and reducing the progression of lifelong complications. Crysvita works by specifically targeting FGF23, restoring phosphate regulation and supporting healthier bone formation. The European Commission’s decision significantly broadens treatment access across the EU and EEA, giving physicians an evidence-based therapeutic option during the earliest stages of disease progression when intervention may provide the greatest long-term clinical benefit.
Clinical Study Supports Regulatory Approval
The EC approval is supported by findings from the Phase 1/2 BUR-CL207 clinical study, an open-label, multicenter trial evaluating the safety, tolerability, pharmacokinetics, and efficacy of burosumab in infants from birth to one year of age diagnosed with XLH. Clinical results demonstrated that the treatment maintained a safety profile consistent with previous studies conducted in older pediatric patients and adults while providing encouraging evidence supporting early disease management. Regulatory authorities concluded that the benefit-risk profile supported extending treatment to the youngest patient population affected by XLH. In addition to expanding the approved indication, the decision grants Crysvita a two-year extension of orphan market exclusivity within the European Union, extending regulatory protection until February 2030. This additional exclusivity recognizes the medicine’s importance in treating a rare disease while encouraging continued innovation in therapies targeting underserved patient populations.
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Rare Disease Innovation Continues to Advance Pediatric Care
The latest regulatory approval further strengthens Kyowa Kirin’s leadership in developing innovative therapies for rare genetic diseases while expanding access to precision medicines for pediatric patients. Since its initial European authorization, Crysvita has become an established treatment option for children, adolescents, and adults living with XLH, with reimbursement already available across several European countries. The expanded infant indication reinforces the growing emphasis on early diagnosis, targeted biologic therapies, and personalized rare disease management, helping clinicians intervene before irreversible skeletal damage occurs. As precision medicine continues transforming rare disease treatment, approvals such as this highlight the importance of combining scientific innovation with earlier clinical intervention to improve quality of life for patients and families. The decision also underscores Europe’s continued support for advancing therapies that address significant unmet medical needs within pediatric rare disease populations.
Source: Kyowa Kirin Press release
