This Market Spotlight report covers the amyloidosis market, comprising key pipeline and marketed drugs, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals.
- The author 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, the author estimates there were approximately 14,600 cases of AL amyloidosis worldwide in 2016.
- Vyndaqel (tafamidis meglumine) is the only US Food and Drug Administration (FDA)-approved drug for transthyretin (TTR)- related hereditary amyloidosis (familial amyloid polyneuropathy).
- There are more industry-sponsored drugs in active clinical development for amyloidosis in Phase III than in any other phase. Therapies in development for amyloidosis focus on targets such as TTR, AL, cluster of differentiation (CD)38, proteasome, and serum amyloid P (SAP) component. The majority of therapies in development for amyloidosis are administered via the intravenous route, with the remainder being oral and subcutaneous formulations.
- High-impact upcoming events for drugs in the amyloidosis space include topline Phase III trial results for patisiran. There were seven licensing and asset acquisition activities involving amyloidosis drugs during 2012-17. The largest deal during that time was the $1,135m exclusive worldwide licensing and development agreement (in August 2012) between Janssen Biotech and Genmab, for the development and commercialization of Genmab's daratumumab and a backup human CD38 antibody.
- The clinical trials distribution across Phases I-IV indicates that the majority of trials for amyloidosis have been in early and midphases 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. The UK leads the major EU markets, while Japan has the top spot in Asia.
- Clinical trial activity in the amyloidosis space is dominated by completed trials. GlaxoSmithKline has the highest number of completed clinical trials for amyloidosis, with four.
- Pfizer has carried out the most Phase III trials in amyloidosis (with three), followed by Alnylam, which has sponsored one Phase III trial.
Key Topics Covered:
Bone marrow/stem cell transplantation
Palliative or supportive care
Amyloidosis in multiple myeloma incidence methodology
Approvals by country
KEY UPCOMING EVENTS
LICENSING AND ASSET ACQUISITION DEALS
GSK Passes On Taking Inotersen To FDA With Ionis
Fortress Subsidiary Licenses Amyloidosis Candidate From Columbia
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
APOLLO Success Clears Alnylam For Lift-Off
Ionis Touts Inotersen's Convenience As Phase III Safety Data Disappoint
Alnylam Plays Up Patisiran As Polyneuropathy Drug Nears Filings
Alnylam Ends Revusiran In Phase III, But Was Failure Due To Safety Or Efficacy?
Ionis Antisense Platform Safety Questioned After Thrombocytopenia Seen In Studies
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