Kyowa Kirin extends Crysvita EU orphan market exclusivity to February 2030

UK— Kyowa Kirin’s European subsidiary, Kyowa Kirin EMEA, has received approval from the European Commission (EC) to expand the use of Crysvita (burosumab) to infants aged one month to one year with X-linked hypophosphataemia (XLH) across the European Union (EU) and European Economic Area (EEA).

The approval follows a positive recommendation issued in April 2026 by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

The regulatory decision allows healthcare professionals to consider burosumab for younger children with XLH, a rare and progressive genetic disorder that can affect skeletal development from infancy.

XLH causes excessive phosphate loss through the kidneys, leading to low phosphate levels in the blood and impaired bone mineralisation.

As a result, affected children may develop skeletal deformities, poor growth and other complications as the disease progresses.

The expanded approval enables physicians to intervene earlier in the disease course, potentially providing treatment options for infants who previously fell outside the approved age range.

Clinical evidence supports expanded indication

The regulatory decision was supported by findings from BUR-CL207, a Phase I/II, open-label, multi-centre clinical trial that evaluated the efficacy, safety, tolerability and pharmacokinetics of burosumab in paediatric patients from birth to one year of age.

According to Kyowa Kirin, the safety profile observed among infants in the study was consistent with the established safety profile of burosumab.

Myriam Hakim, regional franchise head at Kyowa Kirin EMEA, said the approval gives healthcare professionals an opportunity to begin treatment at a younger age.

“This approval means healthcare professionals can now consider treatment with burosumab from as young as one month of age, creating an opportunity to address the disease earlier than ever before. It represents an important step forward for infants living with XLH and the families who care for them,” she said.

Orphan exclusivity extended to 2030

The EC approval also qualifies Crysvita for an additional two years of orphan market exclusivity in the EU for XLH.

This extension will provide regulatory protection for the treatment until February 2030.

Burosumab is a recombinant human monoclonal antibody that targets fibroblast growth factor 23 (FGF23), a protein involved in regulating phosphate levels in the body.

By blocking FGF23 activity, the therapy helps increase phosphate levels and improve phosphate metabolism in people with XLH.

The treatment is reimbursed in several European countries, including France, Germany, Italy, Spain and the UK, for eligible paediatric and adult patients with XLH.

In May 2026, Kyowa Kirin also secured approval from the US Food and Drug Administration (FDA) for an update to Crysvita’s prescribing information.

The update introduced a new dosing option for adults living with XLH.