Voxelotor for haemolytic anaemia treatment gains positive opinion

Global Blood Therapeutics (GBT) has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has awarded a positive scientific opinion–under the Early Access to Medicines Scheme (EAMS)–for voxelotor.

The treatment is an oral once-daily tablet under review by the MHRA for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years-of-age and older. Voxelotor will be offered as monotherapy or in combination with hydroxycarbamide.

The positive opinion means that patients living with SCD–and meeting the eligibility criteria–can gain early-pre-license access to voxelotor, while the MHRA finalises its review of the Marketing Authorisation Application (MAA).

Voxelotor works by increasing haemoglobin’s affinity for oxygen. As oxygenated sickle haemoglobin does not polymerise, while voxelotor inhibits sickle haemoglobin polymerisation and the resultant destruction of red blood cells. GBT believes that voxelotor has the potential to modify the course of SCD. The treatment is approved in the US and has been given the seal of approval through a Priority Medicines (PRIME) designation from the European Medicines Agency.

SCD is a rare inherited blood disorder, affecting approximately 15,000 people in the UK. Patients experience progressive, serious complications and comorbidities, including end-organ damage, which can lead to a decreased quality of life and early death. There is no known cure for SCD, but there are treatments to help manage the condition.

“This decision marks a significant milestone for the sickle cell community in the UK,” said Arvind Agrawal, UK medical director at GBT. “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. GBT is delighted with the progress to help eligible patients have access to this innovation as soon as possible.”