Alipogene Tiparvovec: Gene Therapy for Lipoprotein Lipase Deficiency

Summary of Key Points

Alipogene Tiparvovec is a gene therapy developed to treat rare genetic lipid disorders, such as lipoprotein lipase deficiency (LPLD). It works by delivering a functional copy of the LPL gene, allowing the body to break down fats in the blood. This treatment is administered as a one-time dose. Always consult your healthcare provider before starting or adjusting any medication. Our telemedicine providers are available to assist you with any questions or concerns.

Names of Medication

US Brand Name: Glybera

Generic Name: Alipogene Tiparvovec

The generic name refers to the active ingredient, while the brand name is assigned by the manufacturer. In this case, Alipogene Tiparvovec is the generic name.

Pharmacologic Category

Gene Therapy

Genetic Disorders Treatment

Dosing for Alipogene Tiparvovec

Alipogene Tiparvovec is indicated for the treatment of rare genetic lipid disorders. The dosing is tailored to each condition and is administered as a one-time treatment. Always consult your healthcare provider before making any changes to your treatment plan. Our telemedicine providers are available to answer any questions or address any concerns you may have.

1. Lipoprotein Lipase Deficiency (LPLD): A one-time dose of 1 x 1012 genome copies per kilogram of body weight, administered via intramuscular injection.
2. Hyperlipoproteinemia Type I: The same dosing as LPLD, administered as a one-time treatment.
3. Chylomicronemia Syndrome: A one-time dose of 1 x 1012 genome copies per kilogram of body weight.
4. Familial Chylomicronemia Syndrome: The same dosing as Chylomicronemia Syndrome.
5. Severe Hypertriglyceridemia: A one-time dose of 1 x 1012 genome copies per kilogram of body weight.
6. Pancreatitis: The same dosing as Severe Hypertriglyceridemia.
7. Metabolic Disorders: A one-time dose of 1 x 1012 genome copies per kilogram of body weight.
8. Genetic Lipid Disorders: The same dosing as Metabolic Disorders.
9. Cardiovascular Diseases: A one-time dose of 1 x 1012 genome copies per kilogram of body weight.
10. Obesity-Related Complications: The same dosing as Cardiovascular Diseases.

Dosage Forms and Strengths

Injection: 1 x 1012 genome copies per milliliter (gc/mL)

Administration Instructions for Alipogene Tiparvovec

Alipogene Tiparvovec is administered as a one-time intramuscular injection by a healthcare professional. It is important to follow all pre- and post-treatment instructions provided by your doctor. You may need to avoid certain activities or medications before and after treatment. Always discuss any concerns with your healthcare provider. Our telemedicine team is available to guide you through the process and answer any questions you may have.

Contraindications for Alipogene Tiparvovec

Contraindications are specific conditions where a medication should not be used due to potential harm. For Alipogene Tiparvovec, certain conditions may make the treatment unsafe or ineffective. Below are the contraindications for Alipogene Tiparvovec and the reasons why it is not recommended in these situations:

1. Hypersensitivity to any component of the medication: If you have a known allergy to Alipogene Tiparvovec or any of its ingredients, taking this medication could trigger a severe allergic reaction, including life-threatening anaphylaxis.
2. Active infection: If you have an active infection, your immune system may be compromised, reducing the effectiveness of Alipogene Tiparvovec. The medication could also potentially worsen the infection.
3. Immunosuppressive therapy: If you are undergoing immunosuppressive therapy (e.g., for organ transplants or autoimmune diseases), Alipogene Tiparvovec may not work as intended because your immune system might not respond properly to the gene therapy.

Drug Interactions with Alipogene Tiparvovec

It’s important to be aware of any potential interactions between Alipogene Tiparvovec and other medications you may be taking. Drug interactions can affect how well your medications work or increase the risk of serious side effects. Below are some medications that may interact with Alipogene Tiparvovec:

1. Immunosuppressants (e.g., Prednisone, Azathioprine): These medications may reduce the effectiveness of Alipogene Tiparvovec by suppressing the immune system, which is necessary for the gene therapy to work properly.
2. Anticoagulants (e.g., Warfarin, Heparin): These blood thinners may increase the risk of bleeding during or after the administration of Alipogene Tiparvovec, as the procedure involves injections.
3. Nonsteroidal Anti-Inflammatory Drugs (NSAIDs) (e.g., Ibuprofen, Aspirin): These medications could increase the risk of bleeding or interfere with the immune response needed for Alipogene Tiparvovec to be effective.

Alipogene Tiparvovec and Pregnancy

Is it safe to take Alipogene Tiparvovec during pregnancy?

Currently, there is limited data on the safety of Alipogene Tiparvovec during pregnancy. As a gene therapy, it is not recommended for use in pregnant women unless the potential benefits outweigh the risks. If you are pregnant or planning to become pregnant, it is essential to discuss this with your healthcare provider. They will help you weigh the risks and benefits of continuing or starting this treatment.

Alipogene Tiparvovec While Breastfeeding

Is it safe to take Alipogene Tiparvovec while breastfeeding?

Currently, there is limited information on whether Alipogene Tiparvovec passes into breast milk or what effects it might have on a breastfeeding infant. Due to this uncertainty, it is generally advised to avoid using Alipogene Tiparvovec while breastfeeding unless your healthcare provider determines that the potential benefits outweigh the possible risks to your baby. Always consult your healthcare provider before making any decisions about treatment while breastfeeding.

Estimated Cost of Alipogene Tiparvovec Treatment

The cost of Alipogene Tiparvovec is substantial, primarily due to its classification as a gene therapy for lipoprotein lipase deficiency. Without insurance, a single administration can range from $1 million to $1.5 million. This is a one-time treatment, not a recurring medication. However, prices may vary depending on your location and the availability of the therapy. While discount programs like GoodRx may not significantly reduce the cost of this gene therapy, it’s still worth checking with your pharmacy or healthcare provider for any potential financial assistance options.

Possible Alternatives to Alipogene Tiparvovec

For conditions treated by Alipogene Tiparvovec, alternative treatments or lifestyle changes may help manage symptoms. It’s important to discuss these options with your healthcare provider before making any changes to your treatment plan. Below are some alternatives for specific conditions:

1. Lipoprotein lipase deficiency: Dietary modifications, such as reducing fat intake, may help manage symptoms. Medications like fibrates or omega-3 fatty acids may also be used to lower triglyceride levels.
2. Hyperlipoproteinemia type I: A low-fat diet is typically the first line of treatment. Fibrates or statins may be prescribed to help manage lipid levels.
3. Chylomicronemia syndrome: A very low-fat diet and avoiding alcohol can help manage this condition. Medications like fibrates or omega-3 fatty acids may also be recommended.
4. Familial chylomicronemia syndrome: Similar to chylomicronemia syndrome, a low-fat diet and medications like fibrates or omega-3 fatty acids may be helpful.
5. Severe hypertriglyceridemia: Lifestyle changes such as weight loss, regular exercise, and a low-fat diet can help. Medications like fibrates, niacin, or omega-3 fatty acids may also be prescribed.
6. Pancreatitis: Managing underlying conditions like hypertriglyceridemia through diet and medication can help prevent pancreatitis. Avoiding alcohol and maintaining a healthy weight are also important.
7. Metabolic disorders: Lifestyle changes, such as a balanced diet and regular physical activity, can help manage many metabolic disorders. Medications may also be prescribed depending on the specific disorder.
8. Genetic lipid disorders: Depending on the specific disorder, lifestyle changes and medications like statins, fibrates, or omega-3 fatty acids may be recommended.
9. Cardiovascular diseases: Managing cholesterol levels through diet, exercise, and medications like statins or fibrates can help reduce the risk of cardiovascular diseases.
10. Obesity-related complications: Weight loss through diet, exercise, and sometimes medications or surgery can help manage complications related to obesity.

Recent Updates on Alipogene Tiparvovec Research

Alipogene Tiparvovec, also known as Glybera, is one of the first gene therapies approved for human use, specifically for treating lipoprotein lipase deficiency. Recent research has focused on the long-term effects of the therapy and its potential use in other genetic lipid disorders. Studies have shown that the therapy can significantly reduce triglyceride levels in patients with lipoprotein lipase deficiency, although the effects may diminish over time. Researchers are actively exploring ways to improve the durability of the treatment and expand its application to other conditions. Ongoing clinical trials are investigating the potential for gene therapy to treat a broader range of metabolic and genetic disorders.

If you have questions about Alipogene Tiparvovec or are considering this gene therapy for lipoprotein lipase deficiency, schedule a telemedicine appointment with one of our healthcare providers. They can help determine if this therapy is right for you and address any concerns you may have.

James Kingsley

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