What is Rivfloza prescribed for?
Rivfloza is used to treat Late-Onset Pompe Disease, a rare condition where the body can’t break down sugar properly due to a missing enzyme.
This causes sugar to build up in the muscles, leading to muscle weakness and breathing problems. Rivfloza helps by replacing the missing enzyme, making it easier for the body to manage the condition and reduce symptoms.
What is the name of the drug and what does it do?
Rivfloza, pronounced Riv-flo-za, (generic name: nedosiran) is a newly FDA-approved medication designed for adults with late-onset Pompe disease who are experiencing symptoms despite existing enzyme replacement therapies.
Pompe disease causes muscle weakness and trouble breathing because of the buildup of a sugar called glycogen in cells.
Rivfloza helps reduce this buildup and improve muscle and respiratory function.
How does Rivfloza work?
Rivfloza is an enzyme replacement therapy, meaning it supplements the body with a version of the enzyme that is deficient in those with Pompe disease.
The enzyme provided by Rivfloza helps break down excess glycogen in muscle and other tissues, preventing its harmful accumulation and aiding in restoring muscle function and overall cellular health.
Administered via an IV, Rivfloza directly targets the enzyme deficit at the core of this condition.
What did the research discover?
The approval of Rivfloza was based on results showing that it significantly improves symptoms related to Pompe disease, such as muscle strength and respiratory function.
The clinical trials demonstrated that when Rivfloza was used alongside another treatment called Opfolda, patients experienced marked improvements in their ability to perform daily activities and their quality of life.
These benefits were measured using various medical tests that assess muscle and respiratory health.
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 the infusion (such as rash, hives, or fever)
- Increased risk of infections due to potential immune system suppression
- Respiratory issues, such as coughing or difficulty breathing
What are the dosage recommendations and how is it prescribed?
Rivfloza is administered as an intravenous infusion every two weeks.
It must be given in a healthcare setting under the supervision of a medical professional to monitor for adverse reactions and ensure the correct dosage.
Patients are also pre-treated with Opfolda about one hour before each Rivfloza infusion to enhance the effectiveness of the treatment.
Source:
- Highlights of Prescribing Information for [Drug Name from the first link]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf. Accessed 17 July 2024.
- Drug Trials Snapshots: RIVFLOZA. FDA. Available from: https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza. Accessed 17 July 2024.
