This Market Spotlight report covers the Amyloidosis market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming and regulatory events, probability of success, and a 10-year disease incidence forecast, as well as presenting drug-specific revenue forecasts.
Key Takeaways
Key Takeaways
- The publisher 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 multiple myeloma cases, the publisher estimates there were approximately 19,300 incident cases of AL amyloidosis worldwide in 2017.
- Approved drugs in the amyloidosis space target cluster of differentiation 38 and transthyretin. These drugs are administered via intravenous, subcutaneous, and oral routes.
- There are equal numbers of industry-sponsored drugs in active clinical development for amyloidosis in Phase I and Phase III. 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, 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, Onpattro, CAEL-101, and AKCEA-TTR-LRx, and an expected supplemental CHMP opinion for Darzalex Faspro.
- The overall likelihood of approval of a Phase I metabolic-other asset is 7.9%, and the average probability a drug advances from Phase III is 70.6%. Drugs, on average, take 9.0 years from Phase I to approval, compared to 9.1 years in the overall metabolic space.
- 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 75% of trials in Phase I–II, and 25% in Phase III–IV.
- The US leads in terms of the number of amyloidosis clinical trials globally. The UK leads the major European markets, while Japan has the top spot in Asia.
- GlaxoSmithKline, Bristol Myers Squibb, and Pfizer have the highest number of completed clinical trials for amyloidosis, with five trials each.
- GlaxoSmithKline leads industry sponsors with the highest overall number of clinical trials for amyloidosis.
Table of Contents
OVERVIEWKEY TAKEAWAYSMARKETED DRUGSPIPELINE DRUGSKEY UPCOMING EVENTSPROBABILITY OF SUCCESSREVENUE OPPORTUNITYBIBLIOGRAPHYAPPENDIX
DISEASE BACKGROUND
TREATMENT
EPIDEMIOLOGY
RECENT EVENTS AND ANALYST OPINION
KEY REGULATORY EVENTS
CLINICAL TRIAL LANDSCAPE
LIST OF FIGURES
LIST OF TABLES
