Market Spotlight: Waldenström Macroglobulinemia (WM)

This Market Spotlight report covers the Waldenström Macroglobulinemia market, comprising key marketed and pipeline drugs, clinical trials, 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 2016, there were approximately 4,600 incident cases of Waldenström macroglobulinemia in people aged 40 years and over worldwide, and forecasts that number to increase to 6,000 incident cases by 2025. 

It is estimated that the majority of diagnosed cases worldwide were in males in 2016. 
Worldwide, the incidence of Waldenström macroglobulinemia is highest among individuals aged 60–79 years. Imbruvica, a small molecule tyrosine kinase inhibitor, is the only drug approved by the US Food and Drug Administration for Waldenström macroglobulinemia. 

The majority of industry-sponsored drugs in active clinical development for Waldenström macroglobulinemia are in Phase II, with only one drug in Phase III. 

Therapies in mid-to-late-stage development for Waldenström macroglobulinemia focus on targets such as the PI3K/AKT pathway, proteasome, Bruton’s tyrosine kinase, and cluster of differentiation 20. These drugs are administered via the oral, intravenous, and subcutaneous routes. 


There have been only two licensing and asset acquisition deals involving Waldenström macroglobulinemia drugs during 2013–18. The $50m collaboration agreement between Genmab and Novartis, pursuant to which Novartis intends to transition Arzerra (ofatumumab) for the treatment of certain chronic lymphocytic leukemia indications, was the largest deal. 

Velcade’s US parent product patent is set to expire in July 2022, which will open the door to generic entry. The distribution of clinical trials across Phase I–IV indicates that almost all trials for Waldenström macroglobulinemia have been in the early and mid-phases of development, with 96% of trials in Phase I–II, and only 4% in Phase III. 

The US has a substantial lead in the number of Waldenström macroglobulinemia clinical trials globally. France leads the major EU markets, while China has the top spot in Asia. 

Clinical trial activity in the Waldenström macroglobulinemia space is dominated by completed trials. Takeda, Celgene, and Novartis have the highest number of completed clinical trials for Waldenström macroglobulinemia, with 11, six, and five trials, respectively. 

Takeda leads industry sponsors with the highest number of clinical trials for Waldenström macroglobulinemia, followed by Celgene.