Transthyretin amyloidosis is caused by transthyretin, which is generated by the liver and forms dimers before becoming monomers. Monomers combine to create amyloid fibrils, which are found in organs like the heart, neurological system, gastrointestinal tract, and kidneys. FAP is a kind of hereditary transthyretin amyloidosis, with the Val30Met variation of Transthyretin being the most prevalent cause (TTR). In familial amyloid polyneuropathy, symptoms appear when the patient reaches the age of 30, but they might appear as early as 20 years or as late as 80 years. The symptoms of peripheral neuropathy and autonomic neuropathy vary based on their location. In the case of excess amyloid protein accumulating in the nerves, symptoms may worsen.
The worldwide transthyretin amyloidosis treatment market is estimated to be worth US$ 35.8 million in 2018 and to grow at a 55.4 percent CAGR during the forecast period (2018–2026).
Major market companies have innovative medicines in the pipeline that are in late-stage clinical studies and are likely to be approved soon. Eidos Therapeutics, Inc., for example, is developing AG10, an orally given, small drug designed to potently and selectively stabilise tetrameric TTR, therefore interfering with processes that lead to ATTR. The medication is presently being tested in a Phase 2 clinical study.
Akcea Therapeutics Inc. and Ionis Pharmaceuticals are working together to develop AKCEA-TTR- LRx, a drug that inhibits the synthesis of transthyretin. Akcea Therapeutics Inc. is working to develop AKCEA-TTR-LRx for individuals suffering from both hereditary and wild-type forms of the illness. AKCEA-TTR-LRx is expected to begin clinical trials in 2018. Key market players are focusing on strategic alliances to increase their market share. For example, Alnylam Pharmaceuticals, Inc. collaborated with Orsini Healthcare, a speciality pharmacy, in August 2018 to provide ONPATTRO (patisiran) lipid complex injectable.