BRIEF—Early access to new sickle cell disease therapy in UK

London-based Global Blood Therapeutics (GBT) has been given a positive scientific opinion under the UK’s Early Access to Medicines Scheme (EAMS), for its sickle cell disease candidate voxelotor.

Marketed in the USA as Oxbryta, the oral, once-daily therapy is currently under review by the British medicines regulator for the treatment of hemolytic anemia due to sickle cell disease.

As a result of the positive opinion, people in the UK who meet the eligibility criteria can gain access while the regulatory review is ongoing.

Medical director Arvind Agrawal said: “The EAMS positive scientific opinion is a key step forward to meeting our goal of providing patients in the UK with the first oral treatment option that inhibits red blood cell sickling and has the potential to reduce acute and chronic complications of sickle cell disease.”