EMA Approves Geron's MAA For Imetelstat To Treat Myelodysplastic syndromes

Geron Corporation, a late-stage clinical biopharmaceutical company, announced that the European Medicines Agency (EMA) has approved imetelstat, a first-in-class investigational telomerase inhibitor, for the treatment of transfusion-dependent anaemia in patients with lower risk myelodysplastic syndromes (MDS). The MAA was filed earlier this month and is currently being reviewed by the European Committee for Medicinal Products for Human Use (CHMP) under the centralized system, which applies to all 27 EU member countries as well as Iceland, Norway, and Liechtenstein.

The MAA will be reviewed in about 14 months.“The EMA validation of the marketing authorization application for imetelstat brings us one step closer to potentially offering this first-in-class therapeutic to lower risk MDS patients in the EU suffering from anaemia,” said John A. Scarlett, M.D., Geron’s chairman and chief executive officer. “Based on the clinical profile of imetelstat to date, we are optimistic about its potential to become a standard of care and address longstanding unmet needs of lower risk MDS patients.” The MAA is based on results from IMerge phase 3, in which the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (p<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders.

Mean haemoglobin levels in imetelstat-treated patients increased significantly (p<0.001) over time compared to placebo patients. Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category, as per pharmabiz. Patient-reported outcomes (PRO) data revealed that imetelstat-treated patients saw a sustained considerable reduction in tiredness when compared to placebo. The most prevalent significant adverse events were short-lived, treatable cytopenias, which was consistent with past imetelstat clinical experience.