This Market Spotlight report covers the amyloidosis market, comprising key marketed and pipeline drugs, key upcoming events, patent information, a 10-year disease incidence forecast, and licensing and acquisition deals.

Key Takeaways

• Datamonitor Healthcare estimates that in 2016, there were approximately 59,000 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 14,600 cases of AL amyloidosis worldwide in 2016.
• Vyndaqel was the first therapy to gain approval in the EU for TTR-related hereditary amyloidosis (familial amyloid polyneuropathy). The drug is not approved in the US, where it received a complete response letter from the FDA in 2012. Vyndaqel stabilizes the TTR protein, preventing dissociation of the native TTR tetramer into monomers, a process implicated in TTR amyloidosis, resulting in the inhibition of TTR amyloid fibril formation.
• The majority of industry-sponsored drugs in active clinical development for amyloidosis are in Phase II, with two drugs in NDA/BLA phase.
• Therapies in development for amyloidosis focus on targets such as transthyretin, amyloid-light chain, cluster of differentiation 38, proteasome, serum amyloid P component, 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 IIb trial results for GSK2398852 and Phase II trial results for AG10, plus expected CHMP opinions and estimated PDUFA dates for both Patisiran and Tegsedi.
• There have been four licensing and asset acquisition deals involving amyloidosis drugs during 2013–18. The largest deal was the $395.0m exclusive licensing and collaboration agreement signed in April 2016 between Regeneron Pharmaceuticals and Intellia Therapeutics to advance CRISPR/Cas gene-editing technology for in vivo therapeutic development.
• The distribution of clinical trials across Phases I–IV indicates that the majority of trials for amyloidosis have been in the early and mid-phases of development, with 80% of trials in Phase I–II and only 20% in Phase III–IV.
• The US has a substantial lead in the number of amyloidosis clinical trials globally, while the UK leads the major EU markets.
• GlaxoSmithKline has the highest number of completed clinical trials for amyloidosis, with four trials. GlaxoSmithKline also leads industry sponsors with the highest number of overall clinical trials for amyloidosis, followed by Pfizer and Prothena.