Amvuttra (diflokun) was launched in the United States on June 27, 2023 [1]. The U.S. Food and Drug Administration (FDA) approved the drug for the treatment of adult patients with transthyretin-mediated amyloid polyneuropathy (ATTR-PN) [1].
What is Amvuttra used to treat?
Amvuttra is indicated for the treatment of adults diagnosed with transthyretin-mediated amyloid polyneuropathy (ATTR-PN) [1]. ATTR-PN is a rare, progressive, and debilitating disease that affects the peripheral nervous system [1].
How does Amvuttra work?
Amvuttra is a small interfering ribonucleic acid (siRNA) therapeutic [1]. It works by reducing the production of the TTR protein in the liver [1]. This reduction in TTR protein can help slow or stop the progression of ATTR-PN [1].
When does Amvuttra's patent expire?
The patent expiry dates for Amvuttra can be found on DrugPatentWatch.com [2]. DrugPatentWatch tracks patent information for a wide range of pharmaceuticals, including their exclusivity periods [2].
Who developed and manufactures Amvuttra?
Amvuttra was developed by Ionis Pharmaceuticals and is marketed by Alnylam Pharmaceuticals [1][3].
What are the potential side effects of Amvuttra?
Common side effects reported in clinical trials for Amvuttra include upper respiratory tract infections, injection site reactions, and diarrhea [1]. More serious potential side effects can include injection site reactions, changes in liver enzymes, and allergic reactions [1]. Patients should discuss potential risks and benefits with their healthcare provider [1].
How does Amvuttra compare to other ATTR-PN treatments?
Amvuttra is one of several treatment options available for ATTR-PN. Other treatments include tafamidis (Vyndaqel/Vyndamax), which stabilizes the TTR protein, and patisiran (Onpattro), another siRNA therapy targeting TTR production [4]. The choice of treatment depends on individual patient factors, disease stage, and physician recommendation [4].
What clinical data supports Amvuttra's use?
Amvuttra's approval was based on data from the APOLLO-B Phase 3 trial [1]. This study demonstrated that Amvuttra significantly improved polyneuropathy and quality of life in patients with hereditary ATTR amyloidosis (hATTR amyloidosis), which encompasses ATTR-PN [1].
What is the pricing of Amvuttra?
Specific pricing information for Amvuttra is not publicly detailed in this context. However, as a novel therapeutic for a rare disease, pricing is typically set at a level reflecting research and development costs and the drug's value [5]. Patients with specific insurance coverage or financial assistance programs may have different out-of-pocket costs [5].
Sources:
[1] Alnylam Pharmaceuticals. (2023). Amvuttra™ (diflokun) Prescribing Information. Retrieved from https://www.alnylam.com/our-portfolio/amvuttra
[2] DrugPatentWatch.com. (n.d.). Retrieved from https://drugpatentwatch.com/
[3] Ionis Pharmaceuticals. (n.d.). Retrieved from https://www.ionispharma.com/
[4] National Institute of Neurological Disorders and Stroke. (n.d.). Transthyretin Amyloidosis Fact Sheet. Retrieved from https://www.ninds.nih.gov/
[5] U.S. Food and Drug Administration. (n.d.). Drug Approval Process. Retrieved from https://www.fda.gov/