Benzinga | Jun 14, 2021 07:08AM EDT

GBT's Voxelotor is First Sickle Cell Disease Treatment to Receive Promising Innovative Medicine Designation in the UK

Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT), a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments for underserved patient communities, announced today that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom has granted a Promising Innovative Medicine (PIM) designation for voxelotor for the potential treatment of hemolytic anemia in adults and adolescent patients 12 years of age and older with sickle cell disease (SCD).

Following a review by the MHRA, PIM designations are given to promising treatments that are likely to offer a major advantage for patients and are an early indication that the treatment is a promising candidate for the Early Access to Medicines Scheme (EAMS). For the MHRA to grant a PIM designation, the product must meet each of the following three criteria:

* The condition should be life-threatening or seriously debilitating with high unmet need, meaning there is no method of treatment, diagnosis or prevention available, or existing methods have serious limitations.

* The medicinal product is likely to offer major advantage over methods currently used in the UK, based on both non-clinical and clinical data.

* The potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit risk balance.1

SCD is a rare genetic condition which affects approximately 15,000 people in the UK2 and 52,000 people across Europe.3 It has a devastating impact on the lives of those it affects and their families, including serious and life-threatening complications that can lead to organ damage and early death. Despite SCD being the first genetic disease to be examined at a molecular level,4 there remains a lack of urgency to treat SCD, which traditionally affects the African and Caribbean community, and healthcare disparities based on race persist.

"The sickle cell disease community, which for decades has been dramatically underserved, deserves innovative treatments that address the underlying cause of this debilitating disease," said Nigel Nicholls, UK general manager of GBT. "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 UK. This is an important step forward on our journey, and we remain committed to developing novel treatments with the hope of transforming the lives of those living with SCD."

A first-in-class oral, once-daily therapy, voxelotor directly inhibits hemoglobin S polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to the tissues and organs in the body.

Voxelotor is approved in the United States under the trade name Oxbryta(r) for the treatment of SCD in patients ages 12 years and older. GBT previously announced its plans to seek regulatory approval from the European Medicines Agency (EMA) for voxelotor in the treatment of hemolytic anemia in SCD patients ages 12 years and older.

Prior to potential marketing authorization, GBT initiated an early access program for voxelotor in Europe and other regions outside the United States, which enables physicians to use early access regulatory and legal pathways to request voxelotor for the treatment of hemolytic anemia in eligible patients with SCD who do not have access to the medicine as part of a clinical trial. If approved for EAMS, the voxelotor early access program would be further extended to eligible UK patients.