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.
- 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 incident multiple myeloma cases, The publisher 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.
Key Topics Covered:
2. Key Takeaways
3. Disease Background
- Targeted therapy
- Bone marrow/stem cell transplantation
- Palliative or supportive care
- Amyloidosis incidence methodology
- Amyloidosis in multiple myeloma incidence methodology
- TTR amyloidosis
6. Marketed Drugs
7. Pipeline Drugs
8. Recent Events and Analyst Opinion
- NEOD001 for AL Amyloidosis (April 18, 2019)
- CAEL-101 for AL Amyloidosis (January 31, 2019)
- AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018)
9. Key Upcoming Events
10. Key Regulatory Events
- A Runaway Hit In US, Pfizer Gains EU Nod for Vyndaqel In Cardiomyopathy
- A Win For Alnylam In England As NICE Reverses Onpattro Rejection
- Scotland Greenlights More Rare Disease Treatments
- FDA Clears Vyndamax On Priority Timetable With Vyndaqel
11. Probability of Success
12. Licensing and Asset Acquisition Deals
- Alexion Gets Japanese Rights To Eidos ATTR Candidate
13. Parent Patents
14. Clinical Trial Landscape
- Sponsors by status
- Sponsors by phase
- Recent events
- Prescription information
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