FDA Approves Luspatercept for First-Line Treatment of Myelodysplastic Syndromes
Luspatercept, TGF-b ligand fusion trap protein, which stimulates terminal erythroid differentiation to enhance red blood cell production was approved this week by the FDA as first-line treatment for lower-risk MDS patients who may require red blood cell transfusions based on results of the Commands study (phase 3 randomized clinical trial).
This is the first drug to replace the use of erythroid stimulating agents as the first line in lower-risk MDS. The development of those classes of erythroid maturating agents started at Moffitt Cancer Center led by Dr. Rami Komrokji almost a decade ago testing Sotatercept first in class similar compound for treating anemia in lower-risk MDS.
The Moffitt team later led the development and approval of luspatercept in the U.S. in lower-risk MDS patients with ring sideroblasts subtype after erythroid stimulating agents failure based on the MEDALIST study published in New England Journal of Medicine where Dr. Komrokji was co-senior author.
The COMMANDS study published in Lancet medical journal recently compared luspatercept to erythroid stimulating agents as first-line therapy in lower-risk MDS patients who require red blood cell transfusions demonstrating higher and more durable responses leading to the current approval. Dr. Komrokji from Moffitt Cancer Center was one of the lead investigators on the study and a steering committee member.
"We are excited about the FDA approval of luspatercept as first-line option for treating anemia in MDS marking the long waited for change in the paradigm for the management of MDS after several decades where erythroid stimulating agents were the only first treatment option for our patients," said Dr. Komrokji. "This should translate to better outcomes for our patients and we continue to test this drug and similar agents in this class in several clinical trials to further extend their benefits to our patients."
