Global Blood Therapeutics Inc (NASDAQ:GBT) is on the right track as far as the development of its Oxbryta® (voxelotor) tablets is concerned. The tablets earned the company the win for the Rare Impact Award from the National Organization for Rare Disorders (NORD).
NORD announced the award at this year’s Rare Disease and Orphan Products Breakthrough Summit. The event takes place annually although this year’s summit was different in that it was held virtually due to the ongoing coronavirus pandemic. The award represents an important milestone for GBT.
Oxbryta is the first therapy that GBT has developed with a focus on the mechanism that directly inhibits the polymerization of hemoglobin. The polymerization is the main cause of the sickling and death of red blood cells in patients suffering from sickle cell disease (SCD). Oxbryta is currently approved as an SCD treatment for adolescents above 12-years-old and adults in the U.S.
“We are honored to receive this recognition from NORD for developing novel therapeutics that meet the needs of the sickle cell community, which has been underserved for far too long,” stated GBT’s CEO, Dr. Ted W. Love.
The CEO also added in his statement that patients diagnosed with sickle cell disease are the true winners despite the company winning the award. They now have more treatment options that are highly effective. Dr. Love also pointed out that his company will work closely with patients, medical practitioners, and the rare disease community towards improving the lives of people that suffer from SCD.
Why is the Rare Impact Award such a big deal?
The Rare Impact Award from NORD celebrates companies, groups, and individuals that develop products that yield drastic improvements in the lives of patients suffering from rare diseases or disorders. NORD is an independent organization that is committed to encouraging the development of therapies for rare disorders.
The recent Rare Impact Award that GBT received from NORD is not the first time that oxbryta has been recognized publicly for its impact in the SCD community. It was also recently nominated for the Breakthrough Drug of the year by the National Xconomy Awards. The latter is also an annual event that recognizes top companies, individuals, and organizations that make notable contributions to life sciences in the U.S.