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Amvuttra: The First Quarterly Injection for Treating hATTR Amyloidosis

This prescription drug was first approved by the FDA on June 13, 2022, based on data from a single clinical trial, where it was administered to 122 patients suffering from hereditary transthyretin-mediated amyloidosis (hATTR). The trial results concluded that Amvuttra significantly improved neuropathy impairment in patients.

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Amvutra is the first and only injection that is given four times a year to treat polyneuropathy of hATTR amyloidosis in adult patients. 

What Is Amvuttra Used To Treat?

Amvuttra is used to treat nerve damage (polyneuropathy) caused by a hATTR disease. 

What Is Hereditary Transthyretin-Mediated Amyloidosis (hATTR)?

hATTR is a rare but debilitating disorder that affects multiple organs and tissues in your body, including the nervous (nerve), cardiac (heart), and gastrointestinal (digestive) systems. This disease occurs due to mutations in the transthyretin (TTR) gene, which normally codes for a protein called transthyretin (TTR). 

Your liver primarily makes TRR protein, which circulates in your blood to transport thyroid hormone (thyroxine) and vitamin A (retinol) throughout the body. 

However, mutations in the TTR gene cause your liver to make abnormal TTR proteins that are defective and break easily. The broken proteins clump together in clusters and form amyloid deposits. The amyloid deposits, when built up in different parts of your body, cause damage to your organs and nerves, specifically the peripheral nerves. 

How Does It Work?

Amvuttra contains an active component, vutrisiran, which is a type of small interfering ribonucleic acid (siRNA). The siRNA is a short piece of genetic material designed to attach to and stop the mutated TRR gene responsible for producing TRR protein. 

This significantly reduces the production of defective TRR protein, which results in decreased formation of amyloid deposits. 

What Is the Success Rate?

As per clinical trials, Amvuttra significantly improved nerve function and muscle strength in patients who took the medication compared to those who did not. Patients who take this medication may experience improvement in their walking speed, nutritional health, and ability to perform everyday activities. 

Who Can Take Amvuttra?

Patients with stage I and stage II polyneuropathy (nerve damage) hATTR amyloidosis can take Amvuttra. 

Stage I nerve damage is when the patient feels leg weakness but can walk without help. 

In Stage II nerve damage, the patient can still walk but requires support. 

Patients whose disease progresses to stage III can still take this medication if the benefits outweigh the risks. 

What Is the Available Dosage Form and Strength?

Amvuttra is available in 25 mg/0.5 ml in a single-dose pre-filled syringe. It can only be obtained with a doctor’s prescription. 

How Is Amvuttra Given?

Amvuttra is given as a subcutaneous injection (under the skin) once every 3 months by an experienced healthcare provider. It is usually injected around an area of the abdomen, thighs, or upper arm. 

How Long Does It Take To Work?

Following the first dosage, Amvuttra begins to decrease TTR protein, and this effect lasts as long as treatment is sustained. In a clinical investigation of people treated with Amvuttra, 48% of patients regained some nerve function at 18 months.

Common Side Effects

Old man suffering from side effects of Amvuttra

Amvuttra also has some side effects, along with its potential benefits. The most commonly reported side effects that have been seen in over 5% of patients after taking the medication include:

  • Pain in the extremities (arms and legs)
  • Arthralgia (joint pain)
  • Shortness of breath (dyspnea)
  • Low vitamin A levels

What Precautions Should You Take?

Before taking Amvuttra, it is important to consult your healthcare provider and share your current health status and medical history. Be sure to tell your doctor if you are:

  • Pregnant or intending to become pregnant: Since there is no available data on the effects of Amvuttra on an unborn baby, it is important to consult your healthcare provider before taking Amvuttra.
  • Breastfeeding: It is not clear whether Amvuttra passes into breast milk; therefore, you should avoid taking it if you’re breastfeeding.

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What Important Things Should You Know About Amvuttra?

Amvuttra can cause low vitamin A levels. As discussed earlier, TRR protein is responsible for transporting retinol (vitamin A) through the body. Taking Amvuttra can significantly lower the amount of vitamin A in your blood. 
Severe vitamin A deficiency can result in vision problems (e.g., night blindness). Therefore, it is important to take vitamin A supplements with a dosage of 2,500 IU to 3,000 IU every day. 
 

Is Amvuttra a Type of Gene Therapy?

Yes, it is categorized as a gene silencer that works to prevent amyloid accumulation and organ damage by limiting the liver’s ability to produce the defective transthyretin (TTR) protein. This results in lower TTR levels throughout the body.
 

Cost

The cost can be notably high for some patients. For a subcutaneous solution (25 mg/0.5 ml), Amvuttra costs around $125,686 for a supply of 0.5 ml. 
Moreover, if you intend to receive treatment for a year, it might cost you around $572,164.
The cost of Amvuttra can vary depending on the pharmacy you visit. However, if you’re considering Amvuttra, it’s advisable to consult with healthcare providers about potential assistance programs that can help reduce out-of-pocket expenses. Contact AmeriPharma if you are interested in exploring options for financial assistance.
 

Drug Summary

Amvuttra is a drug used to treat nerve damage (polyneuropathy) caused by hATTR illness. The aim of this medication is to prevent the production of defective TRR protein, eventually reducing the formation of amyloid deposits in the nerves and organs. Low vitamin A levels and pain in the legs and arms are some common side effects of Amvuttra and can be treatable. 

Amvuttra (Vutrisiran) is a brand name for a prescription drug called vutrisiran. It is typically prescribed to treat polyneuropathy (multiple nerve damage) caused by a condition called hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. 

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REFERENCES:

  1. Adams, D., Tournev, I. L., Taylor, M. S., Coelho, T., Planté-Bordeneuve, V., Berk, J. L., González-Duarte, A., Gillmore, J. D., Low, S., Sekijima, Y., Obici, L., Chen, C., Badri, P., Arum, S. M., Vest, J., & Polydefkis, M. (2022). Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid, 30(1), 18–26. https://doi.org/10.1080/13506129.2022.2091985
  2. Luigetti, M., Romano, A., Paolantonio, A. D., Bisogni, G., & Sabatelli, M. (2020). Diagnosis and Treatment of Hereditary Transthyretin Amyloidosis (hATTR) Polyneuropathy: Current Perspectives on Improving Patient Care. Therapeutics and Clinical Risk Management, 16, 109-123. https://doi.org/10.2147/TCRM.S219979
  3. The Food and Drug Administration (FDA): Amvuttra (Vutrisiran) Prescribing Information: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/215515s000lbl.pdf
  4. Research, C. F. D. E. A. (2023, November 27). Drug trial snapshots: AMVUTTRA. U.S. Food And Drug Administration. https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trial-snapshots-amvuttra
  5. Amvuttra | European Medicines Agency. (2022). https://www.ema.europa.eu/en/medicines/human/EPAR/amvuttra
  6. Canadian Agency for Drugs and Technologies in Health. (2024). Vutrisiran (Amvuttra). NCBI Bookshelf. https://www.ncbi.nlm.nih.gov/books/NBK602494/
This information is not a substitute for medical advice or treatment. Talk to your doctor or healthcare provider about your medical condition prior to starting any new treatment. AmeriPharma™ Specialty Care assumes no liability whatsoever for the information provided or for any diagnosis or treatment made as a result, nor is it responsible for the reliability of the content. AmeriPharma™ Specialty Care does not operate all the websites/organizations listed here, nor is it responsible for the availability or reliability of their content. These listings do not imply or constitute an endorsement, sponsorship, or recommendation by AmeriPharma™ Specialty Care. This webpage may contain references to brand-name prescription drugs that are trademarks or registered trademarks of pharmaceutical manufacturers not affiliated with AmeriPharma™ Specialty Care.
MEDICALLY REVIEWED BY Dr. Christine Leduc, PharmD

Dr. Christine Leduc, PharmD, was born and raised in Irvine, CA. She attended college at Midwestern University, where she graduated cum laude. The most rewarding part of her job is suggesting lifestyle changes, educating patients on how their medication works, and precepting future pharmacists. Her areas of expertise are customer service and knowledge of specialty medication. Having worked in the service industry in the past, she has gained the customer service skills necessary to understand the needs of her patients. Dr. Leduc is currently precepting students from Marshall B. Ketchum University, University of Kansas, and Midwestern University. In her free time, she enjoys traveling, baking, and gardening. See Author Biography

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