Oncology/Hematology > > Hematology– By 12-2 vote, members discover enhancement in transfusion self-reliance outweighs run the risk of
by Mike Bassett, Staff Writer, MedPage Today March 14, 2024
An FDA panel extremely backed investigational imetelstat as a treatment alternative for transfusion-dependent anemia in grownups with lower-risk myelodysplastic syndromes (MDS) who stopped working on erythropoiesis-stimulating representatives (ESAs) or are disqualified for this basic treatment.
Regardless of issues signed up by FDA personnel ahead of Thursday’s conference of the Oncologic Drugs Advisory Committee (ODAC), the committee members concurred by a 12-2 margin that the transfusion-independence advantage showed in a stage III research study of the first-in-class telomerase inhibitor sufficed to get rid of issues about negative impacts connected with the drug.
“This trial fulfilled its main endpoint,” explained panelist Neil Vasan, MD, PhD, of Columbia University Medical Center in New York City, in discussing his vote in assistance of imetelstat’s advantage.
“It uses a brand-new treatment for some clients who might have no other choices,” he included. “The advantage in enhancement in transfusion self-reliance surpassed the danger of cytopenias in a client population and a blood cancer oncology neighborhood that is well versed in these unfavorable occasions and their management.”
Geron, imetelstat’s designer, is looking for conventional approval for the drug, with its application supported by MDS3001, an international stage III trial that consisted of 178 greatly transfused clients who stopped working on ESAs.
The main result revealed that the group randomized to imetelstat had substantially greater rates of red cell (RBC) transfusion self-reliance for a minimum of 8 weeks compared to the group designated to placebo (39.8% vs 15%, P=0.001). The trial likewise fulfilled the essential secondary endpoint of 24-week transfusion self-reliance (28% vs 3.3%P=0.001).
As kept in mind, occurrence of top-quality thrombocytopenia and neutropenia were greater in the research study arm versus the placebo arm, as was making use of myeloid development elements:
- Grade 3/4 neutropenia: 71% vs 7%, respectively
- Grade 3/4 thrombocytopenia: 65% vs 8%
- Myeloid development elements: 36% vs 3%
“Clinicians can continuously examine the dangers and advantages of the drug, and if it’s not working they can stop it,” stated Jorge Nieva, MD, from the Keck School of Medicine of the University of Southern California in Los Angeles. “The information here suffices … so that they can make their own choice.”
“The long-lasting effects of blood transfusions can not be downplayed,” stated Jacqueline Garcia, MD, of the Dana-Farber Cancer Center in Boston. “Seeing the 25% of clients that might have possible long-lasting advantage beyond the 24 weeks and approximately a year is actually remarkable.”
She likewise kept in mind that a lot of the grade 3/4 cytopenias in the imetelstat-treated group were short-term, and stated she was “impressed by the truth they did not lead to severe infections.”
ODAC Chair Ravi Madan, MD, of the National Cancer Institute in Bethesda, Maryland, among the 2 votes versus the drug,