This Market Spotlight report covers the amyloidosis market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, key upcoming events, probability of success, patent information, a 10-year disease incidence forecast, and licensing and acquisition deals.

Key Takeaways

• Datamonitor Healthcare estimates that in 2017, there were approximately 90,500 incident cases of amyloidosis worldwide, with the highest proportion being the primary (amyloid light-chain; AL) amyloidosis type.
• Among incident multiple myeloma cases, Datamonitor Healthcare estimates there were approximately 19,300 cases of AL amyloidosis worldwide in 2017.
• Pfizer’s Vyndaqel was the first therapy to gain approval in the EU for hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy and for transthyretin amyloid cardiomyopathy (ATTR-CM), wild type or hereditary. Additionally, Vyndaqel and Vyndamax are approved by the FDA for the treatment of ATTR-CM, wild type or hereditary. Alnylam’s Onpattro and Akcea’s Tegsedi are approved in the US for hATTR amyloidosis with polyneuropathy in adults. All of these drugs are designed to block the production of TTR amyloid protein.
• Industry-sponsored drugs in active clinical development for amyloidosis are spread evenly across Phase I and Phase III, with four drugs also in Phase II.
• Therapies in development for amyloidosis focus on targets such as transthyretin, amyloid – light chain, cluster of differentiation 38, angiogenesis, DNA, alpha-synuclein, amyloid beta/amyloid plaques, tau proteins, myosin, and catechol-O-methyltransferase. These therapies are administered via the intravenous, oral, and subcutaneous routes.
• High-impact upcoming events for drugs in the amyloidosis space comprise topline Phase III trial results for AG10 and vutrisiran, and topline Phase II trial results for CK-3773274.
• The overall likelihood of approval of a Phase I metabolic-other asset is 9.9%, and the average probability a drug advances from Phase III is 70.6%. Drugs, on average, take 8.9 years from Phase I to approval in the metabolic-other space, as well as in the overall metabolic space.
• There have been 11 licensing and asset acquisition deals involving amyloidosis drugs during 2015–20. The largest deal was the $1,740m agreement in 2018 between Ionis Pharmaceuticals and Akcea Therapeutics, pursuant to which Akcea was granted an exclusive worldwide license to Ionis’s inotersen and AKCEA-TTR-LRx (formerly IONIS-TTR-LRx).
• The distribution of clinical trials across Phase I–IV indicates that the majority of trials for amyloidosis have been in the early and mid-phases of development, with 71% of trials in Phase I–II, and 29% in Phase III–IV.
• The US has a substantial lead in the number of amyloidosis clinical trials globally. The UK leads the major European markets, while Japan has the top spot in Asia.
• GlaxoSmithKline has the highest number of completed clinical trials for amyloidosis, with five trials.
• GlaxoSmithKline leads industry sponsors with the highest overall number of clinical trials for amyloidosis, followed by Pfizer.