Publish Date:

03/08/2023

Last Review Date:

02/24/2023

Overview

This document addresses the use of Lamzede (velmanase alfa-tycv), an enzyme replacement therapy approved by the Food and
Drug Administration (FDA) for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric
individuals. Alpha-mannosidosis is a rare genetic lysosomal storage disorder caused by a deficiency of the enzyme alpha-mannosidase due to
pathogenic variants in the MAN2B1 gene. The enzyme deficiency leads to progressive accumulation of oligosaccharides with
characteristic disease features including intellectual disability, ataxia, coarse face, hearing loss, skeletal dysplasia and
immunodeficiency. Accurate diagnosis is important to differentiate alpha-mannosidosis from other lysosomal storage disorders and
provide appropriate enzyme replacement therapy. Diagnosis is confirmed through enzymatic activity measurement or genetic testing. Lamzede has a black box warning for severe hypersensitivity reactions including anaphylaxis. Appropriate medical support measures,
including cardiopulmonary resuscitation equipment, should be readily available during Lamzede administration. If a severe
hypersensitivity reaction occurs, Lamzede should be discontinued immediately, and appropriate medical treatment should be initiated. In individuals with severe hypersensitivity reaction, a desensitization procedure to Lamzede may be considered.

Clinical Criteria

When a drug is being reviewed for coverage under a member's medical benefit plan or is otherwise subject to clinical review (including
prior authorization), the following criteria will be used to determine whether the drug meets any applicable medical necessity
requirements for the intended/prescribed purpose.

Lamzede (velmanase alfa-tycv)

Initial requests for Lamzede (velmanase alfa-tycv) may be approved if the following criteria are met:

1. Individual has a diagnosis of alpha-mannosidosis; AND
2. Documentation is provided that diagnosis is demonstrated by one of the following (Malm 2019):
   • Deficiency in alpha-mannosidase enzyme activity as measured in fibroblasts or leukocytes; OR
   • MAN2B1 gene mutation; AND
3. Individual is using for the treatment of non-central nervous system disease manifestations.

Continuation requests for Lamzede (velmanase alfa-tycv) may be approved if the following criterion is met:

1. There is clinically significant improvement or stabilization in clinical signs and symptoms of disease (including but not limited to
   improvement in motor function, improvement in pulmonary function, reduction in serum oligosaccharides) compared to the
   predicted natural history trajectory of disease.

Lamzede (velmanase alfa-tycv) may not be approved when the above criteria are not met and for all other indications.

Quantity Limits

Lamzede (velmanase alfa-tycv) Quantity Limit

1

Lamzede (velmanase alfa-tycv) 10 mg vial

1 mg/kg once per week

Coding

The following codes for treatments and procedures applicable to this document are included below for informational purposes. Inclusion
or exclusion of a procedure, diagnosis or device code(s) does not constitute or imply member coverage or provider reimbursement
policy. Please refer to the member's contract benefits in effect at the time of service to determine coverage or non-coverage of these
services as it applies to an individual member.

HCPCS

J0217

ICD-10 Diagnosis

E77.1

Injection, velmanase alfa-tycv, 1 mg [Lamzede]

Defects in glycoprotein degradation

Document History

New: 02/24/2023