The FDA has approved pacritinib for myelofibrosis patients with low platelet counts.
Investigators at the Mays Cancer Center, home to UT Health San Antonio MD Anderson Cancer Center, played a lead role in gaining U.S. Food and Drug Administration approval of pacritinib to treat the chronic leukemia known as myelofibrosis. The FDA granted accelerated approval of the drug yesterday. The oral capsule is the first therapy specifically approved for myelofibrosis patients with low platelet counts, a condition called thrombocytopenia.
Ruben Mesa, MD, FACP, executive director of Mays Cancer Center, served as principal investigator of the PERSIST 1 Global Phase III trial of pacritinib. “The Mays Cancer Center has been a leading site in the design and conduct of the final trials involved with the drug’s approval,” Dr. Mesa said Tuesday.
Dr. Mesa has led or co-led development of four drugs for chronic leukemia over the past 11 years:
- Ruxolitinib for myelofibrosis, approved in 2011.
- Ruxolitinib for polycythemia vera, approved in 2014.
- Fedratinib for myelofibrosis, approved in 2019.
- Besremi (ROPEG INF) for polycythemia vera, approved in 2021.
- Pacritinib (Vonjo) for myelofibrosis, approved in 2022.
“Pacritinib is an important addition to the armamentarium we have for treating patients with myelofibrosis, in particular because of the safety and effectiveness that has been seen for a subset of patients with myelofibrosis who have low platelet counts,” Dr. Mesa said in a video on the Cancer Network website.
Patients with low platelets can have difficulties with enlarged spleen (splenomegaly), lower than normal numbers of blood cells (cytopenia), or risk of progressing to acute leukemia. These conditions may impact survival. About 21,000 patients in the U.S. have myelofibrosis and about a third of them suffer from thrombocytopenia.
“Our amazing clinical research team is essential for these trials to succeed,” Dr. Mesa said. “Dr. Adolfo Diaz Duque leads our hematology clinical trials, and Cindy McKeown serves as director of research operations. On behalf of all South Texas patients with myelofibrosis who will benefit from pacritinib therapy, we thank the highly expert and diligent professionals in our Hematology Trial Clinical Research Unit.”