Oxbryta Given Promising Innovative Medicine Status in UK

Steve Bryson, PhD avatar

by Steve Bryson, PhD |

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A Promising Innovative Medicine (PIM) designation has been granted to Oxbryta (voxelotor), a treatment to reduce the destruction of red blood cells in people, 12 and older, with sickle cell disease (SCD).

The designation, given to promising therapies that are likely to provide major benefit to patients, follows a review by the Medicines and Healthcare Products Regulatory Agency in the U.K.

To receive this designation, products must seek to treat life-threatening or seriously debilitating medical conditions for which there is a high unmet need; be likely superior to other treatments currently available in the U.K.; and provide benefits that outweigh potential adverse side effects.

“The sickle cell disease community, which for decades has been dramatically underserved, deserves innovative treatments that address the underlying cause of this debilitating disease,” Nigel Nicholls, U.K. general manager of Global Blood Therapeutics (GBT), the therapy’s developer, said in a press release.

“Voxelotor is the first SCD treatment to receive the PIM designation, and this is a significant milestone in our efforts to potentially make this therapy available in the U.K,” he added.

In general, therapies that receive the PIM designation are potential candidates for an Early Access to Medicines Scheme (EAMS), which allows certain patients to have early access to new therapies prior to their approval, upon request of their physicians.

GBT initiated an early access program for Oxbryta in Europe and other areas outside the U.S. to treat those who do not have access to the medication as part of a clinical trial. With EAMS approval, the early access program would extend Oxbryta to eligible patients, 12 and older, in the U.K.

People with SCD carry a mutation in the HBB gene, which provides instructions for making hemoglobin, the protein inside red blood cells that transports and delivers oxygen to tissues and organs.

Oxbryta is a first-in-class, once-a-day, oral medication that blocks the formation of rigid, long rods (polymers) of faulty hemoglobin molecules that deform red blood cells into a crescent or sickle-like shape, eventually destroying them.

The medication increases hemoglobin’s affinity to oxygen, effectively preventing rod formation, along with red blood cell sickling and their subsequent destruction.

Oxbryta was granted accelerated approval in the U.S. to treat SCD patients, 12 and older, and is also under regulatory review in Europe for the same indication.

As a condition of U.S. approval, GBT will continue to evaluate Oxbryta in the HOPE-KIDS 2 study (NCT04218084), a post-approval confirmatory Phase 3 trial to assess the therapy’s ability to lower the risk of strokes in children, 2–14 years old, with SCD. The trial is enrolling patients at sites worldwide.

Recently, data from the ongoing Phase 2 HOPE-KIDS 1 trial (NCT02850406) showed that six months of treatment with Oxbryta effectively and safely increased hemoglobin levels and reduced hemolysis (destruction of red blood cells) in children, ages 4—11, with SCD. GBT expects these positive results will help support Oxbryta’s future expansion filing to allow younger children in the U.S. to have access to the therapy.

These findings and other data, including real-world evidence supporting the therapy’s use in patients 12 and older are being shared across six poster and oral presentations at the European Hematology Association 2021 Virtual Congress, running June 9–17.

In the U.S., Oxbryta was given fast track, orphan drug, breakthrough therapy, and rare pediatric disease designations for the treatment of SCD. European regulatory authorities named the therapy an orphan drug and granted it a priority medicines designation. All these designations are meant to support the therapy’s development and regulatory review.