The European Medicines Agency (EMA) has granted an orphan drug status to US-based clinical-stage biotechnology company GlycoMimetics' GMI-1070 (rivipansel sodium), designed to treat vaso-occlusive crisis (VOC) in patients with sickle cell disease.
GlycoMimetics and Pfizer are developing the GMI-1070, which earlier received orphan drug and fast track status from the US Food & Drug Administration.
Pfizer will execute next steps of clinical development for the new drug.
GlycoMimetics vice president of clinical development and chief medical officer Dr Helen Thackray noted the company is extremely pleased that the EMA has granted orphan drug status to GMI-1070 for patients with sickle cell disease experiencing VOC.
"Given that people living with sickle cell disease currently do not have enough treatment options, we appreciate all regulatory efforts to help bring the drug to market quickly, assuming that additional studies will continue to demonstrate the potential for GMI-1070 to benefit those living with the disease," Thackray added.
The orphan drug status approval in the EU, given to products for treatment of rare diseases, will provide the companies with development and commercial incentives.
These development and commercial incentives include a 10-year period of market exclusivity, access to a centralized review process, protocol assistance and scientific advice during product development, waiving or reduction of certain fees, and eligibility for grants and research and development support initiatives.
