Geron announces FDA approval of RYTELO (imetelstat)

The telomerase inhibitor, will be used for the treatment in Adult Patients with Lower-Risk MDS with Transfusion-Dependent Anemia

Geron Corporation biopharma company, announced the U.S. Food and Drug Administration (FDA) approval for RYTELO (imetelstat). The drug is for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

“With the approval and availability of RYTELO, we believe eligible patients with lower-risk MDS can potentially experience meaningful clinical benefit, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer.

Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue (1). These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival (2,3). There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis.

 Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence.

References:

1. Lewis R, Bewersdorf JP, Zeidan AM. Clinical Management of Anemia in Patients with Myelodysplastic Syndromes: An Update on Emerging Therapeutic Options. Cancer Manag Res. 2021 Jan 25;13:645-657. doi: 10.2147/CMAR.S240600. PMID: 33531837; PMCID: PMC7846829.

      2. Cogle CR, Reddy SR, Chang E, et al. Early treatment initiation in lower-risk myelodysplastic syndromes           produces an earlier and higher rate of transfusion independence. Leuk Res. 2017;60:123-128.

      3. Balducci, L. (2006), Transfusion independence in patients with myelodysplastic syndromes. Cancer, 106:          2087-2094. https://doi.org/10.1002/cncr.21860