LUMIZYME® (alglucosidase alfa) Treatment for Pompe
LUMIZYME® is the brand name for alglucosidase alfa, a drug that was approved by the Food and Drug Administration (FDA) on May 25th, 2010 for Late-Onset Pompe Disease and then on August 1st, 2014 for patients of all ages with Pompe Disease.
Frequently Asked Questions About LUMIZYME®
Is my child eligible for treatment with LUMIZYME®?
LUMIZYME® is for patients with patients with Pompe, regardless of genetic mutation. However, you should have a consultation with a physician if your child is CRIM negative, as immune modulation may need to be consider to reduce the likelihood of an infusion reaction.
How does LUMIZYME® work?
It is a medication that replaces a missing or deficient enzyme in people with Pompe disease (acid α-glucosidase (GAA) deficiency). Since people with Pompe disease do not produce enough of the GAA enzyme on their own, a replacement enzyme may be needed. The genetically engineered enzyme is intended to act like the naturally occurring GAA that is normally produced within human cells.
How is LUMIZYME® given to patients?
The enzyme replacement therapy is given to patients intravenously, typically on a bi-weekly timeframe with the infusion lasting around four hours.
What is the benefit of LUMIZYME®?
LUMIZYME® has demonstrated in clinical trials reduced levels of glycogen in muscle biopsies following treatment.
Is LUMIZYME® covered by insurance?
You will get a CareConnectPSS care manager that will help you and your child’s physician better understand your insurance benefits so you can get access to the treatment your child needs.