This Market Spotlight report covers the Waldenström Macroglobulinemia market, comprising key marketed and pipeline drugs, probability of success, clinical trials, recent events and analyst opinion, patent information, a 10-year disease incidence forecast, and licensing and 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, the incidence of WM is highest among individuals aged 60–79 years.
• Imbruvica, a small molecule tyrosine kinase inhibitor, is the only drug approved by the FDA for WM.
• The majority of industry-sponsored drugs in active clinical development for WM are in Phase II, with only one drug in Phase III. 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, p110 delta/PIK3CD, and proteasome. These drugs are administered via the oral, intravenous, and subcutaneous routes.
• High-impact upcoming events for drugs in the WM space comprise an estimated PDUFA date for Imbruvica and an expected CHMP opinion for Brukinsa.
• The overall likelihood of approval of a Phase I hematologic cancer asset is 10%, and the average probability a drug advances from Phase III is 61.6%. Drugs, on average, take 9.1 years from Phase I to approval, compared to 9.3 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 93% of trials in Phase I–II, and only 7% in Phase III–IV.
• The US has a substantial lead in the number of WM clinical trials globally. The UK and France lead the major European markets, while China has the top spot in Asia.
• Takeda has the highest number of completed clinical trials for WM, with 11 trials. Johnson & Johnson leads industry sponsors with the highest overall number of clinical trials for WM, followed by Bristol Myers Squibb and Takeda.