Rare Disease

First and Only Enzyme Replacement Therapy Approved for Rare Alpha-Mannosidosis

  • March 14, 2023
  • < 1 min read

The US FDA has approved Lamzede (velmanase alfa) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare genetic complication characterized by the deficiency of the enzyme alpha-mannosidosis in the body.

The medication Lamzede – velmanase alfa is designed to act the same way as the enzyme AM which is also known as alpha-mannosidosis in the human body thus restoring normal cellular activity in patients.

What is Alpha-mannosidosis:

Alpha-mannosidosis or AM is a rare genetic, progressive lysosomal storage disorder. The manifestations of this genetic disorder may vary, but typically include mild to moderate intellectual disability, impaired immune system, muscle weakness, skeletal abnormalities, distinctive facial features, and hearing loss.

This rare disease (AM) is caused by certain changes in the gene MAN2B1, which codes for the lysosomal alpha-mannosidosis enzyme. 

Mutations of the MAN2B1 are responsible for the deficiency of production of the alpha-D mannosidase enzyme or the production of a defective, inactive form of the enzyme. The rare disorder (AM) affects about 1 in every 500,000 individuals worldwide.

What is Lamzede (velmanase alfa):

Formulated as a recombinant form of human alpha-mannosidase, Lamzede (velmanase alfa) is intended to provide or supplement natural alpha-mannosidase. This medicine is administered via intravenous (IV) infusion once weekly. For Alpha-mannosidosis, the proposed dosage is 10 mg once every week.

The most commonly reported side effects include hypersensitivity reactions including nasopharyngitis, pyrexia, anaphylaxis, headache, and arthralgia.