The market for treating transthyretin amyloidosis in the United States is predicted to expand faster with the introduction of novel medicines. For instance, evidence published by PubMed new in October 2018 indicated that medicines utilising RNA interference also show clinical promise to treat amyloidosis. An experimental RNA interference drug called patisiran precisely prevents the formation of transthyretin in the liver. The U.S. Food and Drug Administration approved the first RNA interference (RNAi)-based medication, patisiran, in 2018. Patisiran is an effective and secure medication for the treatment of hereditary transthyretin amyloidosis, according to results from phase I to III clinical trials (hATTR). It is predicted that patisiran's efficacy, the first siRNA-based medication to receive FDA approval, will be confirmed by real-world data from a wider group of patients who received treatment.
The U.S. Transthyretin Amyloidosis Treatment Market is estimated to be valued at US$ 50.7 million in 2022 and is expected to exhibit a CAGR of 9.0% during the forecast period (2022-2030).
Leading Companies in the U.S. Transthyretin Amyloidosis Treatment Industry:
1. Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.
2. Pfizer, Inc.
Pfizer Inc. was founded in 1849 and is headquartered in New York, New York. Pfizer Inc. discovers, develops, manufactures, markets, distributes, and sell biopharmaceutical products worldwide. In December, 2022 announced the U.S. Food and Drug Administration (FDA) granted Emergency Use Authorization (EUA) of their Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine as the third 3-µg dose in the three-dose primary series for children 6 months through 4 years of age.
3. Prothena Corporation Plc.
Prothena Corporation plc, a late-stage clinical company, focuses on discovery and development of novel therapies for life-threatening diseases. The company was founded in 2012 and is based in Dublin, Ireland.
4. GlaxoSmithKline plc.
GSK plc, together with its subsidiaries, engages in the creation, discovery, development, manufacture, and marketing of pharmaceutical products, vaccines, over-the-counter medicines, and health-related consumer products. GSK plc was founded in 1715 and is headquartered in Brentford, the United Kingdom. GSK plc and Wave Life Sciences Ltd in December, 2022 announced a strategic collaboration to advance oligonucleotide therapeutics, including Wave’s preclinical RNA editing programme targeting alpha-1 antitrypsin deficiency (AATD), WVE-006.
5. Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.
6. Eidos Therapeutics
7. SOM Innovation Biotech, S.L
SOM Biotech established in 2009 is a biopharmaceutical company based in Barcelona, Spain. SOM has an extensive portfolio of products that includes drugs for orphan diseases including TTR Amyloidosis, Huntington's disease, Tardive Dyskinesia, Phenylketonuria, Niemann-Pick C, Glioblastoma and Parkinson’s disease.
*Definition- Transthyretin amyloidosis's current treatment options mostly focus on easing patients' symptoms while also addressing the condition's fundamental cause through a liver donation.
