| Common Brands | Nexviadyme |
|---|---|
|
Drug Class |
Enzyme Replacement Therapy (ERT) |
|
Controlled Substance Classification |
Not a controlled medication |
|
Generic Status |
Lower-cost generic available |
|
Availability |
Prescription only |
Avalglucosidase alfa, known by its brand name Nexviazyme, is a recombinant enzyme replacement therapy aimed at the treatment of Pompe disease. Pompe disease is a rare and often deadly genetic disorder, caused by a deficiency in an enzyme called acid alpha-glucosidase, also known as GAA.
Without sufficient GAA, glycogen starts to pool within the tissues, which in turn causes muscle weakness, breathing problems, and some other severe health concerns.
Avalglucosidase alfa is used to supplement or substitute for this deficient enzyme; it helps in reducing the reaccumulation of glycogen and alleviate the symptoms of this disease.
Avalglucosidase alfa should be given intravenously by the healthcare provider. It is usually prepared for an infusion every two weeks. The rate or dose can be adjusted based on the conditions of the patient and what best suits their treatment.
Monitoring: In terms of monitoring, the patient’s vital signs, like blood pressure, heart rate, and oxygen levels, will be monitored while infusing to ensure that the medication is being well tolerated.
Pre-infusion considerations: Patients will be premedicated with medications such as antihistamines, steroids, etc, to lessen the risk of any allergic reaction. During and after infusion, if any adverse effect ensues, the infusion rate may be changed or some additional intervention may be performed by the health care provider to control side effects.
Avalglucosidase alfa works in the body of the patient with Pompe disease by substituting defective or missing acid alpha-glucosidase. GAA’s function is to break down glycogen into glucose within the lysosomes, which is a waste disposal system for the cell.
When GAA is deficient or missing, glycogen begins to gather in tissues, mainly within muscles, hence turning them into slow-onset breakdowns and dysfunction.
Infusing the enzyme Avalglucosidase alfa helps introduce it into the lysosomes, where it degrades the excess glycogen, thereby reducing glycogen accumulation and resulting in improved muscle function, decreased tissue damage, and some of the debilitating symptoms of Pompe disease.
This slows down the progression of the disease, improves respiratory function and facilitates mobility, thus eventually leading to an improvement in the quality of life for the patient.
Although Avalglucosidase alfa is generally well tolerated, there are some adverse reactions with which patients should be aware:
Infusion-related reactions: These include symptoms such as fever, chills, rash, dizziness, nausea, and headaches. These reactions are usually experienced during or soon after the infusion.
Fatigue: Some patients may suffer from fatigue, particularly after the infusion.
Muscle/joint pains: Mild to moderate muscle or joint pain is another common side effect.
Allergic reactions: Avalglucosidase alfa may cause allergic reactions including but not limited to anaphylaxis. Patients should be observed for any signs or symptoms of an allergic response during and following the infusion. Avalglucosidase alfa should be administered in a medical setting and by individuals experienced in the treatment of severe hypersensitivity reactions.
Heart conditions: Patients with pre-existing heart conditions should be made known to the healthcare provider prior to initiating treatment with Avalglucosidase alfa, due to the risk of cardiovascular side effects.
Liver function: Although rarely observed, liver function ought to monitored in those patients receiving enzyme replacement therapy due to the possibility of presenting issues with the liver.
Pregnancy and breastfeeding: Avalglucosidase alfa has not been well studied in pregnant or breastfeeding women, so discuss with a healthcare provider about whether the benefits of treatment outweigh risks during pregnancy or lactation.
Immunosuppressive Drugs: Avalglucosidase Alfa may increase the risk of allergic reactions or infusion-related reactions if combined with immunosuppressive drugs (e.g., corticosteroids or methotrexate), which affect immune system response.
Vaccines: Receiving live vaccines while undergoing treatment with Avalglucosidase Alfa may increase the risk of infection due to immune system alterations. It’s important to consult a healthcare provider before getting any live vaccines.
Other Enzyme Replacement Therapies: Combining Avalglucosidase Alfa with other enzyme replacement therapies may lead to an increased risk of adverse effects, such as immune reactions or complications with treatment effectiveness, depending on the condition being treated.
The usual dose for Avalglucosidase alfa is based on the patient’s weight. Most patients receive a dose of
Initial dose: Avalglucosidase alfa is given at 20 mg/kg every two weeks. The starting dose can be adjusted based on the patient’s response and tolerance to treatment.
Dose adjustment in case of infusion reactions: In cases of severe infusion-related reactions, dose or rate may be adjusted
Dosage and administration should be followed strictly according to the health care provider’s prescription
Avalglucosidase alfa is a prescription-only drug and is only to be prescribed by a physician, often a specialist like a neurologist or geneticist. As it is a very rare disease, the diagnosis of Pompe disease should be confirmed with genetic testing or enzyme activity assays prior to initiation of treatment with Avalglucosidase alfa.
Patients should have regular follow-up appointments to monitor the effectiveness of the treatment, manage any potential side effects, and adjust the treatment plan as necessary.
What is Avalglucosidase Alfa?
Avalglucosidase Alfa is a therapeutic enzyme replacement used to treat Pompe disease, a rare genetic disorder caused by the deficiency of the enzyme acid alpha-glucosidase.
How does Avalglucosidase Alfa work?
It works by replenishing the deficient enzyme in patients with Pompe disease, helping to break down glycogen and prevent its accumulation in the muscles and other tissues.
Who is a candidate for Avalglucosidase Alfa treatment?
Candidates typically include individuals diagnosed with Pompe disease, particularly those with the classic infantile-onset or late-onset forms of the disease.
What are the common side effects of Avalglucosidase Alfa?
Common side effects may include infusion-related reactions, respiratory issues, and allergic reactions.
How is Avalglucosidase Alfa administered?
It is administered via intravenous infusion, typically every two weeks, under supervision in a healthcare setting to monitor for any adverse reactions.
Can Avalglucosidase Alfa be used in children?
Yes, Avalglucosidase Alfa can be used in both infants and children diagnosed with Pompe disease, and it may be initiated at any age.
Is Avalglucosidase Alfa effective for all patients with Pompe disease?
While it is effective for many patients, the response can vary based on factors like age at diagnosis and the severity of the disease.
What should patients do if they miss a dose of Avalglucosidase Alfa?
Patients should contact their healthcare provider for guidance on rescheduling missed doses and should not attempt to dose themselves.
How should Avalglucosidase Alfa be stored?
It should be stored refrigerated and protected from light until it is ready to be used. Any unused medication should be discarded.
Are there any contraindications for using Avalglucosidase Alfa?
Patients with known hypersensitivity to Avalglucosidase Alfa or any of its components should not use this medication.
What monitoring is required during treatment with Avalglucosidase Alfa?
Patients require regular monitoring of their clinical status, pulmonary function, and muscle function, as well as monitoring for infusion-related reactions.
Can Avalglucosidase Alfa interact with other medications?
It’s important to inform healthcare providers about all medications being taken, including over-the-counter drugs and supplements, to manage potential interactions.
What is the long-term outlook for patients on Avalglucosidase Alfa?
Long-term treatment may help improve or stabilize muscle function, respiratory function, and overall quality of life, but responses vary.
Is there a risk of developing antibodies to Avalglucosidase Alfa?
Yes, some patients may develop antibodies against the enzyme, which could potentially affect the treatment’s efficacy.
What should I do if I experience an allergic reaction to Avalglucosidase Alfa?
If any signs of an allergic reaction occur (such as rash, itching, or swelling), seek immediate medical attention and inform your healthcare provider.
Are there any dietary restrictions while using Avalglucosidase Alfa?
There are no specific dietary restrictions associated with the use of Avalglucosidase Alfa, but maintaining a balanced diet is essential for overall health.
Is Avalglucosidase Alfa covered by insurance?
Coverage may vary depending on the insurance provider and specific policies. Patients should check with their insurance company regarding benefits.
Where can patients access Avalglucosidase Alfa?
Avalglucosidase Alfa is typically available through specialized pharmacies and clinics that manage enzyme replacement therapies.
What resources are available for support while on Avalglucosidase Alfa?
Patients and families can access support from organizations like the Pompe Foundation and local patient advocacy groups for information and community support.
How can I learn more about ongoing clinical trials for Avalglucosidase Alfa?
Information about clinical trials can be found on websites like ClinicalTrials.gov, as well as through healthcare providers who are familiar with Pompe disease research.
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