What is POMBILITI prescribed for?
POMBILITI is prescribed for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥88 lbs and who are not improving on their
current enzyme replacement therapy (ERT).
What is the name of the drug and what does it do?
POMBILITI, pronounced pom-BILL-ih-tee, (generic name: cipaglucosidase alfa-atga) is a breakthrough treatment approved for adults with late-onset Pompe disease (LOPD) who weigh at least 88 lbs and are not improving on current enzyme replacement therapies.
Pompe disease is a genetic disorder that causes muscle weakness and respiratory issues due to the buildup of a complex sugar called glycogen in the body’s cells.
How does POMBILITI work?
POMBILITI is a treatment designed for Pompe disease, a rare disorder where the body lacks a specific enzyme needed to break down glycogen, a type of sugar that stores energy.
Normally, this enzyme helps convert glycogen into glucose, a form of sugar our bodies use for energy. Without this enzyme, glycogen accumulates, particularly in muscle tissues, leading to muscle weakness and damage that can affect movement and breathing.
To counter this, POMBILITI uses a synthetic version of the missing enzyme, which is administered directly into the bloodstream via an intravenous (IV) infusion.
This synthetic enzyme takes over the role of the missing natural enzyme, helping to break down the excess glycogen. As a result, it reduces the harmful buildup, thereby improving muscle function and overall health in individuals with Pompe disease.
By restoring the enzyme’s activity, POMBILITI not only helps in managing the symptoms but also addresses the underlying cause of the disease, offering significant benefits to those affected by this challenging condition.
What did the research discover?
The FDA approved POMBILITI after studies showed it significantly improved muscle strength and breathing in people with Pompe disease.
During the trials, patients received POMBILITI along with another medication called Opfolda.
This combination was more effective than other treatments, leading to better lung function and muscle strength.
The improvements were measured using tests that check how well muscles and lungs are working, showing that POMBILITI could greatly enhance life quality for patients.
These studies included a wide range of participants, ensuring the results would help many people with this condition.
What are some of the side effects?
The side effects listed are from the product manufacturer. As health scientists, we share this information to keep you informed without causing undue worry. Many side effects diminish over time, and not all patients experience them. If prescribed medication, take it as directed and consult your healthcare professional if you have any concerning side effects.
- Allergic reactions during infusion, such as rash, hives, or fever.
- Risk of infections due to the immune system’s potential suppression.
- Respiratory issues like coughing or trouble breathing, which require close monitoring.
What are the dosage recommendations and how is it prescribed?
POMBILITI is administered every two weeks as an intravenous infusion(IV). The treatment must be performed in a healthcare setting by a professional to monitor for adverse reactions and ensure proper dosage.
Patients also take one dose of Opfolda orally approximately one hour before each POMBILITI infusion to enhance the treatment’s effectiveness.
Source:
- Highlights of Prescribing Information. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761204s000lbl.pdf. Accessed 17 July 2024.
- Drug Trials Snapshots: POMBILITI. FDA. Available from: https://www.fda.gov/drugs/development-approval-process-drugs/drug-trials-snapshots-pombiliti. Accessed 17 July 2024.
