Novo Nordisk said the acquisition, which will give it access to Forma’s lead drug candidate etavopivat, is aligned with its strategy to complement and accelerate its presence and pipeline in hemoglobinopathies, a group of disorders in which there is abnormal production or structure of the hemoglobin protein in the red blood cells.

“By adding Forma’s differentiated approach to address unmet needs for patients, we are taking a step forward in enhancing our sickle cell disease pipeline,” said Ludovic Helfgott, executive vice president and head of rare disease at Novo Nordisk. “We have an ambition to build a leading portfolio with standalone and combination treatments to tackle the complications and underlying causes of sickle cell disease.”

Etavopivat is an oral, once-daily, selective pyruvate kinase-R (PKR) activator, which is being developed to improve anemia and red blood cell health in sickle-cell patients. About 17 million people worldwide live with sickle cell disease, which is a seriously debilitating, life-threatening and life-shortening illness.

The therapy, which is currently under evaluation in two separate trials (Phase 2/3 and Phase 2), has been granted Fast Track, Rare Pediatric Disease and Orphan Drug designations by the US Food and Drug Administration, as well as Orphan Drug designation by the European Medicines Agency.

Author: Elaine Burridge, Freelance Journalist