Comanche Biopharma Corp. announced that the European Medicines Agency (EMA) has granted orphan drug designation for CBP-4888 for the treatment of sFlt1-mediated preterm preeclampsia. A serious obstetric complication, preeclampsia is estimated to impact approximately four in 10,000 pregnancies in the European Union each year. A novel siRNA drug candidate, CBP-4888 is designed to downregulate overexpression of the protein sFlt1, a known pathogenic mediator of preeclampsia, in the placenta. Currently an investigational drug candidate, CBP-4888 has the potential to be the first precision medicine for an obstetrical indication.
“We are grateful to the EMA for granting orphan designation to CBP-4888 and recognizing preeclampsia as a life-threatening, significant unmet medical need,” said Allison August, M.D., Chief Medical Officer at Comanche Biopharma. “Beyond the acute morbidity and mortality risk that preeclampsia presents for mothers and their babies, it is also associated with serious, lifelong health complications. By targeting a root cause of preeclampsia with a precision medicine, our goal is to provide a therapeutic solution that can prevent the far-reaching, short- and long-term impact of this condition for mothers, babies, families and society at large.”
The EMA grants orphan drug designation to drugs and biologics intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that affects fewer than five in 10,000 people in the European Union, and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease. Sponsors who obtain orphan drug designation benefit from protocol assistance, market exclusivity and regulatory fee reductions.
About sFlt1 and CBP-4888
Overexpression of the protein sFlt1 is believed to be a key pathogenic mediator of preeclampsia. When produced in excess by the placenta, sFlt1 is toxic, severely damaging the mother’s blood vessels and impairing the growth of new ones. This overproduction of sFlt1 and subsequent vascular damage result in the common maternal signs and symptoms of preeclampsia and in fetal growth restriction.
CBP-4888 is designed to achieve sufficient uptake by the placenta to selectively downregulate the disease-causing overexpression of the sFlt-1 protein in patients with sFlt-1-mediated preterm preeclampsia. CBP-4888 is being investigated for the potential to ameliorate the acute, potentially life-threatening maternal symptoms of preterm preeclampsia. This, in turn, may enable the safe prolongation of pregnancy and continued fetal maturation, thereby minimizing the near-term morbidity and mortality for mothers and their babies, and the potential long-term consequences for both.
In addition to receiving Orphan Drug Designation by the EMA, CBP-4888 has also received Fast Track Designation by the U.S. Food and Drug Administration, and Innovation Passport Designation by the U.K. Innovative Licensing and Access Pathway (ILAP) Steering Group.
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