The European Commission has approved an expanded indication for Crysvita (burosumab) to treat infants with X-linked hypophosphataemia (XLH) from 1 month to 1 year of age across the European Union and European Economic Area, allowing much earlier intervention in the rare, progressive bone disease. The decision broadens existing EU approvals and opens access to therapy for the youngest XLH patients, whose skeletal development can be affected from infancy.
XLH is a lifelong genetic disorder characterized by phosphate wasting that impairs bone mineralization, leading to skeletal deformities, impaired growth, and other serious complications, with clinical signs often emerging during infancy or early childhood. Earlier diagnosis and treatment are considered critical to limit disease progression and protect long-term skeletal development and physical function.
Kyowa Kirin EMEA said the new approval will enable healthcare professionals to consider burosumab treatment from as early as one month of age. Regional Franchise Head Myriam Hakim called the decision an important step forward for infants and families affected by XLH because it creates an opportunity to address the disease “earlier than ever before.”
The EC ruling follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in April 2026 and is supported by data from BUR-CL207 (NCT04188964), a Phase 1/2 open-label, multicenter study in pediatric patients from birth to one year with XLH. Investigators reported that the safety profile observed in infants was consistent with the established safety profile of burosumab.
The decision also grants Crysvita a two-year extension of orphan market exclusivity for XLH in the EU, extending regulatory protection from February 2028 to February 2030.
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