Imetelstat, a first-in-class telomerase inhibitor from Geron Corporation (NASDAQ:GERN) is on its way to becoming a potential treatment for myelodysplastic syndromes (MDS). The company says it had received a positive opinion on the application for orphan drug designation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Previously, Imetelstat had received orphan drug designation from the FDA.
How Does A Medicine Qualify For An Orphan-Drug Status?
Myelodysplastic syndromes (MDS) are a group of disorders. They mainly occur when there is a defect from the blood-forming cells in the bone marrow. According to the Mayo Clinic, this is considered cancer in some cases, hence the need for a bone marrow transplant or chemotherapy.
Meanwhile, the qualification of an orphan-drug status for whatever medicine does not happen. The medicine must have shown the potential and the intention of treating extremely devastating or severe conditions. Additionally, the conditions should be affecting fewer than five in 10,000 people. Upon the product’s approval, the designation gets incentives, the most notable one being 10 years of market exclusivity.
Other benefits include access to a compacted marketing approval procedure, clinical protocol assistance, reduced regulatory fees, and distinguished valuation processes for Health Technology Assessments in selected regions.
According to EMA, Imetelstat did demonstrate the potential of being an advantage to patients with low-risk MDS. This was backed up and affirmed by data from a phase 2 trial. The drug is now in by data from a Phase 3 clinical trial, christened IMerge, and will enroll 170 patients. Geron CEO John Scarlett says they are optimistic that it will achieve the suitability for global regulatory filings.
More Global Sites for the Imerge Phase 3 Clinical Trial
By the end of March, Geron had 66% of operational sites for the IMerge Phase 3 clinical trial. This was in the United States, Asia, the Middle East, and Europe. However, it has anticipation of opening another 90 sites globally.
On the other hand, the company is also very cautious about its employees’ well-being and continues to comply with the local ‘stay at home’ measures. It has also halted all business travel, and instead, it is embracing a virtual working policy.