UnitedHealthcare enzyme therapies Coverage Update

Coverage Criteria for Enzyme and Related Therapies

Aldurazyme (laronidase) - Initial/Continuation

Aldurazyme (laronidase) covered when ALL of the following are met

Aldurazyme initial therapy: Diagnosis of any MPS I syndrome (Hurler, Hurler-Scheie, Scheie)

Aldurazyme diagnostic confirmation: Confirmed by deficiency/absence of alpha-L-iduronidase enzyme activity in fibroblasts or leukocytes OR molecular genetic confirmation of pathogenic mutations in the alpha-L-iduronidase gene

Aldurazyme symptom requirement: Presence of clinical signs and symptoms of MPS I (examples: cardiac abnormalities, corneal clouding, dysostosis multiplex, hepatomegaly, restrictive lung disease)

Aldurazyme dosing and authorization: Dosing per FDA labeling; initial authorization up to 12 months; continuation requires prior receipt and documented positive clinical response with reauthorization up to 12 months

Elaprase (idursulfase) - Initial/Continuation

Elaprase (idursulfase) covered when ALL of the following are met

Elaprase diagnostic confirmation: Diagnosis of MPS II confirmed by deficiency of iduronate 2-sulfatase enzyme activity in fibroblasts or leukocytes with normal activity of another sulfatase OR molecular genetic testing showing deletion or mutations in the iduronate 2-sulfatase gene

Elaprase symptom requirement: Presence of clinical signs and symptoms of MPS II (examples: hepatosplenomegaly, skeletal deformities, neurocognitive decline, cardiovascular disorders)

Elaprase dosing and authorization: Dosing per FDA labeling; initial authorization up to 12 months; continuation requires prior receipt and documented positive clinical response with reauthorization up to 12 months

Elfabrio and Fabrazyme (Fabry disease) - Initial/Continuation

Elfabrio (pegunigalsidase alfa-iwxj) and Fabrazyme (agalsidase beta) covered when ALL of the following are met

Fabry diagnostic confirmation: Diagnosis of Fabry disease confirmed by absence/deficiency (<5% of mean) of alpha-galactosidase A activity in leukocytes, dried blood spot, or serum OR molecular genetic testing demonstrating deletion or pathogenic mutations in GLA (galactosidase alpha) gene

Fabry symptom requirement: Presence of clinical signs and symptoms of Fabry disease (examples: acroparesthesias, angiokeratomas, corneal whorls, hypohidrosis, renal disease, exercise intolerance)

Fabry monotherapy: Patient must not receive the therapy in combination with another disease-modifying Fabry therapy (examples: Elfabrio, Fabrazyme, Galafold)

Fabry dosing and authorization: Dosing per FDA labeling; initial authorization up to 12 months; continuation requires prior receipt and documented positive clinical response with reauthorization up to 12 months

Kanuma and Lamzede - Initial/Continuation

Kanuma (sebelipase alfa) and Lamzede (velmanase alfa-tycv) covered when ALL of the following are met

Kanuma diagnostic confirmation: Diagnosis of lysosomal acid lipase deficiency (LAL-D) confirmed by absence/deficiency of lysosomal acid lipase activity by dried blood spot OR molecular genetic testing for pathogenic variants in LIPA gene

Kanuma symptom requirement: Presence of clinical signs and symptoms of LAL-D (examples: abdominal distention, hepatosplenomegaly, liver fibrosis, ascites)

Kanuma dosing and authorization: Dosing per FDA labeling; initial authorization up to 12 months; continuation requires prior receipt and documented positive clinical response with reauthorization up to 12 months

Lamzede diagnostic confirmation: Diagnosis of alpha-mannosidosis confirmed by absence/deficiency (<10% of lab mean) of alpha-mannosidase activity OR molecular genetic testing for pathogenic variants in MAN2B1 gene

Lumizyme and Pompe disease - Initial/Continuation

Lumizyme (alglucosidase alfa) and other Pompe therapies covered when the following are met

Pompe diagnostic confirmation (infantile): Infantile-onset Pompe: absence/deficiency (<1% of lab specific normal mean) of acid alpha-glucosidase (GAA) activity in skin fibroblasts OR molecular genetic testing demonstrating pathogenic variants in the GAA gene

Pompe symptom requirement: Presence of clinical signs and symptoms of Pompe disease (examples: cardiac hypertrophy, respiratory distress, skeletal muscle weakness)

Pompe monotherapy: Patient is not receiving Lumizyme in combination with another disease-modifying enzyme therapy for Pompe disease (examples: Nexviazyme, Pombiliti)

Pompe dosing and authorization: Dosing per FDA labeling; initial authorization up to 12 months; continuation requires prior receipt and documented positive clinical response with reauthorization up to 12 months

Mepsevii (vestronidase alfa-vjbk) - Initial/Continuation

Mepsevii (vestronidase alfa-vjbk) covered when ALL of the following are met

Mepsevii diagnostic confirmation: Diagnosis of MPS VII confirmed by absence/deficiency of beta-glucuronidase enzyme activity in fibroblasts or leukocytes OR molecular genetic confirmation of pathogenic GUSB mutations

Mepsevii symptom requirement: Presence of clinical signs and symptoms of MPS VII (examples: hepatosplenomegaly, joint limitations, airway or pulmonary problems, limited mobility)

Mepsevii dosing and authorization: Dosing per FDA labeling; initial authorization up to 12 months; continuation requires prior receipt and documented positive clinical response with reauthorization up to 12 months

Naglazyme (galsulfase) - Initial/Continuation

Naglazyme (galsulfase) covered when ALL of the following are met

Naglazyme diagnostic confirmation: Diagnosis of MPS VI confirmed by absence/deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase) activity in fibroblasts or leukocytes OR molecular genetic confirmation of pathogenic ASB gene mutations

Naglazyme symptom requirement: Presence of clinical signs and symptoms of MPS VI (examples: kyphoscoliosis, genu valgum, pectus carinatum, gait disturbance, growth deficiency)

Naglazyme dosing and authorization: Dosing per FDA labeling; initial authorization up to 12 months; continuation requires prior receipt and documented positive clinical response with reauthorization up to 12 months

Naglazyme (galsulfase)