Vimizim

Diagnosis is confirmed by enzymatic assay for N-acetylgalactosamine-6-sulfate sulfatase (GALNS) activity in peripheral blood leukocytes or fibroblasts (excluding multiple sulfatase deficiency) AND mutational analysis of GALNS¹; AND 

Patient is under the care of a specialist with experience in the diagnosis and management of MPS IVA; AND 

The following baseline evaluations prior to initiation of Vimizim (elosulfase alfa) must be provided with the request for coverage: 

Detailed medical history documenting surgeries, medical admissions, subspecialty assessments 

Orthopedic evaluation including spinal and cranial MRI, skeletal x-rays, pain symptoms from bone and joints as appropriate to age and clinical disease. 

Mobility measure: 6MWT and stair climb (if appropriate for age and disease status) 

Respiratory function testing including sleep study testing (if appropriate for age) 

Age-appropriate quality of life measure (such as HAQ, PODCI, EQ5D5L or SF36)² 

documentation of mobility aide requirement, such as a walker or cane 

documentation of requirement for respiratory aides, including ventilation status and changes in respiratory support requirements; 

Ophthalmologic and ear, nose and throat (ENT) assessment (if appropriate) 

Urine keratin sulfate (KS) determination: specific KS determination is preferred over total glycosaminoglycans (GAGs) 

Cardiac echocardiogram 

The patient is diagnosed with an additional progressive life limiting condition where treatment would not provide long term benefit (such as cancer or multiple sclerosis) 

The patient has a forced vital capacity (FVC) of less than 0.3 liters and requires continuous ventilator assistance. 

The patient/family is unwilling to comply with the associated monitoring criteria 

The patient/family is unwilling to attend clinics for assessment and treatment purposes