Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Clinical-stage biopharmaceutical firm Global Blood Therapeutics (GBT) has secured orphan drug designation from the US Food and Drug Administration (FDA) for GBT440 to treat sickle cell disease (SCD).
Subscribe to our email newsletter
GBT440 is an oral, once-daily SCD therapy that works by increasing hemoglobin’s affinity for oxygen.
The company is investigating the drug candidate in both healthy subjects and SCD patients in a phase 1/2 clinical trial. Full results are expected to be available in 2016.
The efficacy of GBT440 is being evaluated on abnormal polymerization of deoxy-hemoglobin, the mechanism of red blood cell sickling.
SCD is an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, resulting in the formation of abnormal hemoglobin, dubbed sickle hemoglobin.
GBT CEO Ted Love said: "Receiving orphan drug designation, along with the previously announced Fast Track designation, are important milestones in our regulatory strategy for GBT440 and highlight the FDA’s agreement that the SCD community faces a critical need for new treatments.
"We continue to believe that GBT440 has the potential to become the first mechanism-based and disease-modifying therapeutic for this grievous disease and look forward to sharing full results from our Phase 1/2 trial and potentially initiating a pivotal trial in adult patients with SCD in 2016."
Apart from GBT440, GBT is also undertaking several research and development activities for hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis and acute respiratory distress syndrome, as well as hereditary angioedema.
Image: SCD is an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin. Photo: courtesy of dream designs/FreeDigitalPhotos.net.