burosumab

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Generic Name:
burosumab
Project Status:
Active
Therapeutic Area:
Treatment of X-Linked Hypophosphatemia
Manufacturer:
Kyowa Kirin Canada, Inc.
Call for patient/clinician input open:
Brand Name:
Crysvita
Project Line:
Reimbursement Review
Project Number:
SR0818-000
Call for patient/clinician input closed:
NOC Status at Filing:
Post NOC
Biosimilar:
No
Manufacturer Requested Reimbursement Criteria1:
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).
Submission Type:
Reassessment
Fee Schedule:
Schedule A
Indications:
For the treatment of X-linked hypophosphataemia (XLH) in adult patients.
  1. The requested reimbursement criteria are provided by the applicant and do not necessarily reflect the views of CADTH. Reimbursement criteria from CADTH will be documented in the final recommendation, if applicable.

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Key Milestones2
Call for patient/clinician input openNovember 14, 2023
Call for patient/clinician input closedJanuary 12, 2024
Clarification:

- Patient input submission received from Canadian XLH Network

Submission receivedJanuary 02, 2024
Submission acceptedJanuary 16, 2024
Review initiatedJanuary 17, 2024
Draft CADTH review report(s) provided to sponsor for commentApril 04, 2024
Deadline for sponsors commentsApril 15, 2024
CADTH review report(s) and responses to comments provided to sponsorMay 09, 2024
Expert committee meeting (initial)May 22, 2024
Draft recommendation issued to sponsorJune 04, 2024
Draft recommendation posted for stakeholder feedbackJune 13, 2024
End of feedback periodJune 27, 2024