Kyowa Kirin secures extended EC approval for CRYSVITA in X-linked hypophosphataemia treatment

Kyowa Kirin has recently received expanded approval from the European Commission (EC) for its recombinant monoclonal antibody treatment, CRYSVITA (burosumab), in the management of X-linked hypophosphataemia (XLH). This extended approval now includes the treatment of older adolescents and adults, significantly broadening the patient population eligible for this breakthrough therapy. Previously, CRYSVITA was only approved for children aged one year and older, with radiographic evidence of bone disease, and for adolescents with growing skeletons.

What Is X-linked Hypophosphataemia and Why Is CRYSVITA Crucial?

X-linked hypophosphataemia (XLH) is a rare, inherited condition that typically manifests in early childhood, leading to severe, progressive symptoms such as lower limb deformities, stunted growth, and chronic bone and joint pain. It is caused by a genetic defect that impairs phosphate reabsorption in the kidneys, leading to abnormal bone mineralization. As a life-long condition, XLH often results in complications that impact mobility and overall quality of life, making early and effective treatment essential.

CRYSVITA, a fully human monoclonal IgG1 antibody, works by targeting the underlying pathophysiology of XLH. It helps increase phosphate reabsorption in the kidneys, which is critical for normal bone development and mineralization. This therapy has proven to be a game-changer for children with XLH, and with the new EC approval, adults and older adolescents can now benefit from its life-changing effects.

Expanded Patient Group: Who Now Qualifies for CRYSVITA Treatment?

The expanded approval means that any adolescent with radiographic evidence of bone disease, regardless of growth status, is eligible for treatment. In addition, adults with XLH are now able to access CRYSVITA, offering them a potential solution to manage their symptoms and improve their quality of life. This extended approval is a significant milestone for both patients and healthcare providers, providing an effective treatment option where there were previously few alternatives.

Abdul Mullick, President of Kyowa Kirin International, highlighted the importance of this decision, noting that it marks a significant advancement in the management of XLH, a condition that profoundly affects both children and adults. He emphasized Kyowa Kirin’s commitment to meeting the unmet medical needs of patients and their families, adding that the company is focused on ensuring broad access to CRYSVITA for this expanded patient group.

The Science Behind CRYSVITA’s Effectiveness

The extended approval for CRYSVITA is based on the positive results of two pivotal Phase 3 clinical studies: the UX023-CL303 study, which evaluated CRYSVITA’s efficacy in adults with XLH, and the UX023-CL304 study, which examined its impact on osteomalacia (bone softening) in adults with the condition. These studies demonstrated that CRYSVITA not only helps correct phosphate deficiencies but also improves bone mineralization, reducing pain and enhancing mobility.

The studies’ findings underscore CRYSVITA’s role as the only therapy that targets the underlying cause of XLH. This approval is particularly significant as it addresses the needs of adults, a group that has long been underserved by current treatment options.

Collaboration Between Kyowa Kirin and Ultragenyx Pharmaceutical

Kyowa Kirin has been working in partnership with Ultragenyx Pharmaceutical in the global development and commercialization of CRYSVITA. This collaboration has been instrumental in bringing this innovative therapy to market, ensuring it reaches as many patients as possible around the world.

The partnership reflects a shared commitment to advancing the treatment landscape for rare diseases like XLH. As more patients gain access to CRYSVITA, both companies remain dedicated to ensuring that XLH patients experience better health outcomes and improved quality of life.

A New Hope for XLH Patients

With this extended approval, CRYSVITA stands as a crucial treatment for those suffering from X-linked hypophosphataemia, offering new hope to adolescents and adults who previously had limited options. The expanded patient group eligible for treatment marks a significant step forward in the management of XLH, providing access to a therapy that not only alleviates symptoms but also targets the root cause of the disease. As Kyowa Kirin continues to push for broader access, CRYSVITA remains a critical advancement in the fight against this rare and debilitating condition.