burosumab

In addition to the criteria currently recommended by CADTH in the pediatric population of XLH patients (May 2020), the sponsor is requesting the following criteria for adult patients living with XLH: Initiation Criteria Aligned with international consensus guidelines, burosumab treatment can be initiated in adult patients, who have: a clinical presentation consistent with XLH, including: fasting hypophosphatemia, and normal renal function (defined as fasting serum creatinine below the age-adjusted upper limit of normal), and a confirmed PHEX gene variant in either the patient or in a directly related family member with appropriate X-linked inheritance and persistent bone and/or joint pain due to XLH, and/or osteomalacia that limits daily activities, and/or pseudofractures or osteomalacia-related fractures, and insufficient response or refractory to conventional therapy or if patients experience complications related to conventional therapy. Renewal Criteria Patients should be assessed on an annual basis for continued benefit. Treatment with burosumab can be renewed as long as the patient does not meet any of the discontinuation criteria. Discontinuation Criteria In adult patients burosumab should be discontinued if any of the following occur: hyperparathyroidism, nephrocalcinosis, evidence of fracture or pseudofracture based on radiographic assessment, or intolerable adverse events (e.g., one patient discontinued Study CL303 because of restless leg syndrome worsening from baseline).

For the treatment of X-linked hypophosphataemia (XLH) in adult patients.