This Market Spotlight report covers the Waldenström Macroglobulinemia (WM) market, comprising key marketed and pipeline drugs, probability of success, clinical trials, upcoming events, recent events and analyst opinion, a 10-year disease incidence forecast, and licensing and asset acquisition deals, as well as presenting drug-specific revenue forecasts.

Key Takeaways

• Datamonitor Healthcare estimates that in 2017, there were approximately 5,330 incident cases of Waldenström macroglobulinemia (WM) in people aged 40 years and over worldwide, and forecasts that number to increase to 6,920 incident cases by 2026.
• It is estimated that the majority of diagnosed cases worldwide were in males in 2017.
• Worldwide, incident cases of WM are highest among individuals aged 60–79 years. BeiGene’s Brukinsa, AbbVie’s Imbruvica, and Gilead’s tirabrutinib are the only approved drugs available for WM. These therapies are administered via the oral route.
• The majority of industry-sponsored drugs in active clinical development for WM are in Phase II, with one drug in the NDA/BLA stage.
• Therapies in development for WM focus on targets such as B-cell lymphoma 2 (Bcl-2)/Bcl-2 family, Bruton’s tyrosine kinase, chemokine receptor 4, lipid rafts, casein kinase 1 epsilon, PI3K/AKT pathway, and p110 delta/PIK3CD. These drugs are administered via the oral and intravenous routes.
• High-impact upcoming events for drugs in the WM space comprise an expected CHMP opinion as well as a PDUFA date for Brukinsa.
• The overall likelihood of approval of a Phase I hematologic cancer asset is 8.5%, and the average probability a drug advances from Phase III is 58.4%. Drugs, on average, take 9.5 years from Phase I to approval, compared to 9.6 years in the overall oncology space.
• The distribution of clinical trials across Phase I–IV indicates that almost all trials for WM have been in the early and mid-phases of development, with 94% of trials in Phase I–II, and only 6% in Phase III–IV.
• The US has a substantial lead in the number of WM clinical trials globally. France and the UK lead the major European markets, while China has the top spot in Asia.
• Clinical trial activity in the WM space is dominated by completed trials. Takeda has the highest number of completed clinical trials for WM, with 11 trials.
• Bristol Myers