The FDA approved Geron’s imetelstat to treat certain anemic myelodysplastic syndrome (MDS) patients who are dependent on blood transfusions.
The approval comes after the FDA’s Oncologic Drugs Advisory Committee voted 12 to 2 in March that the benefits of the drug outweigh the risks. Geron’s stock $GERN shot up nearly 20% following Thursday’s approval.
The approval is based on positive results from Geron’s IMerge trial, a 178-person Phase 3 study which evaluated the length of time participants went without needing a red blood cell infusion. The trial demonstrated significantly higher rates of blood transfusion independence in people taking imetelstat, marketed as Rytelo, compared to the placebo.
The drug will be priced at $2,471 for a 47 mg vial and $9,884 for a 188 mg vial, a Geron spokesperson told Endpoints News. The recommended dosage is 7.1 mg/kg administered every four weeks.
Faye FellerFaye Feller, chief medical officer at Geron, said in an interview with Endpoints that there is an unmet need for a treatment that targets the underlying disease in addition to treating the anemia itself.
She explained that undergoing frequent blood transfusions is burdensome for lower risk MDS patients and can lead to other risks like iron buildup. She said lowering dependence on blood transfusions is key to improving quality of life.
“The long term consequences of transfusions, even though they are effective at temporarily relieving anemia, it’s still that whole burden,” she said.
Drug pricing watchdog the Institute for Clinical and Economic Review concluded in a report last month that the drug’s estimated cost of about $250,000 per year was not cost-effective.
The most common adverse reactions included decreased platelets, decreased white blood cells, and Covid-19 infections, among others.
The product was granted orphan designation by the FDA.