Eligible Patients in UK Granted Early Access to Oxbryta

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by Steve Bryson, PhD |

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Oxbryta (voxelotor), a daily oral therapy for hemolytic anemia associated with sickle cell disease (SCD), is now available to eligible patients in the U.K. under an early access program.

It received a positive scientific opinion by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) under the Early Access to Medicines Scheme (EAMS).

“This decision marks a significant milestone for the sickle cell community in the UK,” Arvind Agrawal, UK Medical Director at Global Blood Therapeutics (GBT), Oxbryta’s developer, said in a press release. “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.”

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“GBT is delighted with the progress to help eligible patients have access to this innovation as soon as possible,” Agrawal added.

Oxbryta was granted accelerated approval in the U.S. in 2019 to treat SCD patients, 12 and older. This was recently extended to include children as young as 4, who now also have access to a new therapy formulation that’s more suitable for young children.

GBT’s marketing approval application for Oxbryta is currently under review in Europe, where the company launched a similar early access program for patients who have no alternative treatment options. The therapy is also approved in the United Arab Emirates for individuals 12 and older.

Last year, the therapy was named a Promising Innovative Medicine by the MHRA, a designation that is given to promising therapies that are likely to provide a significant benefit to patients in the U.K.

The goal of EAMS is to grant early access to medicines with this designation, before they’re approved, to patients living with seriously debilitating or life-threatening conditions in the U.K.

Medicines under EAMS that eventually gain approval and receive a positive assessment by the National Institute of Health and Care Excellence may benefit from accelerated commissioning by the National Health Service — a process of examining healthcare needs and planning, prioritizing, and monitoring health services to get the best outcomes.

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In SCD, faulty hemoglobin — the protein inside red blood cells that carries oxygen — clumps together, resulting in the sickling and destruction of red blood cells, causing hemolytic anemia, a common SCD symptom.

Oxbryta is a first-in-class oral therapy designed to bind to hemoglobin and increase its affinity to oxygen, preventing hemoglobin clumping and the associated sickling and loss of red blood cells.

By treating the underlying cause of SCD, the therapy may help restore normal red blood cell function and oxygen delivery to tissues, lessening anemia and the risk of vaso-occlusive crises, which are episodes of pain caused by sickled red blood cells blocking blood flow.

SCD affects an estimated 15,000 people in the U.K. and 52,000 in Europe.