Elosulfase alfa Brand Name– Vimizim
What is Elosulfase alfa
Elosulfase alfa is a purified human enzyme for the treatment of patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
It is produced by recombinant technology in a Chinese hamster ovarian cell line and is administered as a weekly infusion.
MPS IVA is an autosomal recessive lysosomal storage disorder that is caused by a mutation in the gene encoding N-acetylgalactosamine-6-sulfatase (GALNS), which leads to the absence or marked reduction of GALNS activity. Elosulfase alfa is intended to provide exogenous N-acetylgalactosamine-6-sulfatase.
MPS IVA is a multisystem disorder that causes significant functional limitations and reduced quality of life, although the progression of symptoms is variable. Over time, patients have less endurance and more severe exercise and respiratory capacity limitations.
Typically, patients appear unaffected at birth. For severely affected patients, symptoms occur within the first year of life, whereas other patients may not develop initial symptoms until later in childhood or adolescence.
Prior to the availability of elosulfase alfa as an enzyme replacement therapy, treatment for MPS IVA primarily focused on pain management, treatment of infection and respiratory disease, and corrective orthopedic surgeries.
In a 24-week, randomized, double-blind, placebo-controlled trial (n = 176, ages 5—57 years), a statistically significant improvement in the 6-minute walk test was noted in the treatment vs. the placebo group (primary endpoint); however, no difference was found in the rate of stair climbing (secondary endpoint).
Patients receiving elosulfase alfa once weekly walked about 22.5 meters farther in 6 minutes compared with those who received placebo (p = 0.0174). Risk of anaphylaxis is a significant concern with elosulfase alfa.
Premedication with antihistamines with or without antipyretics is recommended, and a careful infusion rate titration schedule must be observed. Patients should be monitored closely during and after the administration of the infusion.
The FDA approved elosulfase alfa in February 2014.
Indications
- mucopolysaccharidosis IVA (Morquio A syndrome)
For the treatment of mucopolysaccharidosis IVA (Morquio A syndrome)
NOTE: Elosulfase alfa has been designated as an orphan drug for this indication by the FDA.
Side Effects
- abdominal pain
- antibody formation
- chills
- cough
- cyanosis
- dyspnea
- erythema
- fatigue
- fever
- flushing
- headache
- hypotension
- nausea
- peripheral edema
- rash
- serious hypersensitivity reactions or anaphylaxis
- spinal cord compression
- urticaria
- vomiting
Monitoring Parameters
- laboratory monitoring not necessary
Contraindications
- breast-feeding
- fever
- geriatric
- infants
- neonates
- pregnancy
- respiratory infection
- serious hypersensitivity reactions or anaphylaxis
- sleep apnea
Interactions
There are no drug interactions associated with Elosulfase alfa products.
