CHMP positive for Rytelo (imetelstat) for transfusion-dependent (TD) anemia due to very low, low or intermediate risk myelodysplastic syndromes (LR-MDS).- Geron Corporation

Geron Corporation  announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of Rytelo (imetelstat) for the treatment of adult patients with transfusion-dependent (TD) anemia due to very low, low or intermediate risk myelodysplastic syndromes (LR-MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. The European Commission (EC), which has the authority to approve medicines in the European Union (EU), will review the CHMP’s recommendation and is expected to make a final decision on the marketing authorization application (MAA) in the following months.

 "If approved, Rytelo would be the first and only telomerase inhibitor available in Europe,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. 

As part of its review of the marketing authorization application (MAA), the CHMP looked at the results from the IMerge Phase III clinical trial and assessed that the benefit of Rytelo in patients with transfusion-dependent anemia due to very low, low or intermediate risk MDS is a reduction in the need for red blood cell transfusions in the first 24 weeks of treatment compared to placebo, as observed in the double-blind controlled study. The most common side effects were thrombocytopenia, leukopenia, neutropenia, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), increased alkaline phosphatase (ALP), asthenia and headache.

About Lower-Risk Myelodysplastic Syndromes (LR-MDS); 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 . 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 . 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 independenceMyelodysplastic syndromes (MDS) are a type of rare blood cancer where you don't have enough healthy blood cells It's also known as myelodysplasia. There are many different types of MDS. Some types can stay mild for years and others are more serious. MDS can affect people of any age, but is most common in adults over the age of 70.