Decision Resources, one of the world's leading research and advisory firms for pharmaceutical and healthcare issues, finds that, through 2018, five new drugs will launch for the treatment of myelodysplastic syndromes (MDS), three of which are expected to offer much-needed second-line therapeutic options in the hypomethylating agent (HMA)-refractory higher-risk MDS population. Currently, there is only one curative treatment for MDS -- allogeneic hematopoietic stem cell transplant (HSCT). However, old age and poor general performance status classifies approximately 75 percent of patients as ineligible for this treatment.
Between 2004 and 2006, the U.S. MDS market expanded when the first drug therapy options were approved for the indication -- azacitidine (Celgene's Vidaza), lenalidomide (Celgene's Revlimid) and decitabine (Eisai/MGI Pharma's Dacogen). No new therapies have been approved in the United States for MDS since 2006; only azacitidine has been approved for MDS in Europe, although experts believe an EU approval for lenalidomide is imminent.
According to Niche Markets and Rare Diseases: Myelodysplastic Syndromes, azacitidine is the leading therapeutic option for the disease in both the U.S. and Europe. Decision Resources anticipates expansion of MDS therapeutic options with the approval of five drugs between 2014-2018: oral azacitidine for transfusion-dependent lower-risk MDS; Onconova/Baxter's intravenous formulation of rigosertib (Estybon) for HMA-refractory higher-risk MDS; Merck's vorinostat (Zolinza) for combination therapy with azacitidine in higher-risk MDS; Cyclacel's oral sapacitabine for HMA-refractory higher-risk MDS; and GlaxoSmithKline's eltrombopag (Revolade) for HMA-refractory higher-risk MDS, with a focal goal of reducing thrombocytopenia. Onconova also has an oral formulation of rigosertib in development for lower-risk patients.
"The near-term MDS drug market will grow modestly as lenalidomide obtains approval in Europe, but the next wave of noteworthy market growth will come between 2014 and 2018," said Decision Resources Vice President Kim Crowell . "While experts acknowledge that the approvals of azacitidine and lenalidomide were groundbreaking, they also report significant remaining unmet need in MDS. Citing the dearth of second-line options for higher-risk MDS patients, experts are hopeful that several Phase II and III drugs will provide valuable alternative therapeutic options, although they do not expect any drug during the next decade to completely shift or uproot the MDS therapeutic landscape. Improving overall survival, without sacrificing quality of life, is the key therapeutic goal in higher risk MDS patients, while functional quality of life via hematologic improvement is the primary goal in lower-risk patients."