- The author estimates that in 2016, there were 46,000 prevalent cases of myelofibrosis in adults aged 50 years and older worldwide, and forecasts that number to increase to 57,000 prevalent cases by 2025.
- Global prevalence of myelofibrosis is estimated to be 0.0027%.
- A majority of industry-sponsored drugs in active clinical development for myelofibrosis are in Phase II. Incyte’s Jakafi (ruxolitinib phosphate) is the only marketed drug for myelofibrosis. It is the first FDA-approved oral JAK1/2 inhibitor for the treatment of the disease.
- Therapies in mid- and late-stage development for myelofibrosis focus on targets such as JAK/STAT and hedgehog signaling pathways, FMS-like tyrosine kinase 3, IL-1 receptor-associated kinase 1, histone deacetylase, telomerase, transforming growth factor-beta receptor, Src kinase family, monocytes, interferon receptor, IL-3 receptor, and BCR-ABL fusion protein. The majority of pipeline drugs in mid- to late-stage development for myelofibrosis are administered via the oral route.
- High-impact upcoming events for drugs in the myelofibrosis space include pacritinib’s topline Phase III trial results and its expected Committee for Medicinal Products for Human Use (CHMP) opinion.
- There were only three licensing agreements during 2013-17. The largest deal during this period was the $1,250m agreement between Bristol-Myers Squibb and Promedior, pursuant to which Bristol-Myers Squibb was granted an exclusive right to acquire Promedior along with its lead asset PRM-151, which is in development for idiopathic pulmonary fibrosis and myelofibrosis.
- The clinical trials distribution across Phase I-IV indicates that the majority of trials for myelofibrosis have been in early and midphases of development, with 89% of trials in Phase I-II, and only 11% in Phase III-IV.
- The US has a substantial lead in the number of myeloproliferative neoplasm clinical trials globally, yet the forecasted prevalent cases of myelofibrosis are lower in North America as compared with Asia and Europe. Germany leads the major EU markets, while South Korea has the top spot in Asia, where the prevalent cases of myelofibrosis are highest.
- Clinical trial activity in the myeloproliferative neoplasms space is dominated by completed trials. Novartis has the highest number of completed clinical trials for myeloproliferative neoplasms.
2 DISEASE BACKGROUND
5 MARKETED DRUGS
6 PIPELINE DRUGS
7 KEY UPCOMING EVENTS
8 KEY REGULATORY EVENTS
- Misery In Myelofibrosis: Pacritinib Setback A Boost For Jakafi
10 PARENT PATENTS
11 REVENUE OPPORTUNITY
12 CLINICAL TRIALS LANDSCAPE
- Sponsors by status
- Sponsors by phase
- Recent events
- Prescription information:
List of Figures
Figure 1: Trends in prevalent cases of myelofibrosis, 2016-25
Figure 2: Pipeline drugs for myelofibrosis in the US
Figure 3: Pipeline drugs for myelofibrosis, by company
Figure 4: Pipeline drugs for myelofibrosis, by drug type
Figure 5: Pipeline drugs for myelofibrosis, by classifications
Figure 6: Key upcoming events in myelofibrosis
Figure 7: Licensing and asset acquisition deals in myelofibrosis, 2012-17
Figure 8: Parent patents in myelofibrosis
Figure 9: Clinical trials in myelofibrosis
Figure 10: Top 10 drugs for clinical trials in myelofibrosis
Figure 11: Top 10 companies for clinical trials in myelofibrosis
Figure 12: Trial locations in myelofibrosis
Figure 13: Myelofibrosis trials status
Figure 14: Myelofibrosis trials sponsors, by phase
List of Tables
Table 1: Prevalent cases of myelofibrosis, 2016-25
Table 2: Marketed drugs for myelofibrosis
Table 3: Approvals by country for myelofibrosis
Table 4: Pipeline drugs for myelofibrosis in the US
Table 5: Historical global sales, by drug ($m), 2012-16
Table 6: Forecasted global sales, by drug ($m), 2017-22