Global Blood Therapeutics (NASDAQ: GBT) has announced that it has received a scientific opinion from the UK Medicines and Healthcare products Regulatory Agency under Early Access to Medicines Scheme for voxelotor, an oral once a day tablet under study by the MHRA for hemolytic anemia resulting from sickle cell diseases treatment in pediatric patients above 12 years and adults as a single agent or a combination with hydroxycarbamide.
A favorable verdict implies SCD patients can get early access of voxelotor
This favorable verdict means that individuals with SCD who fulfill the eligibility requirements can get voxelotor early, pre-license access. At the same time, the MHRA completes its examination of the Marketing Authorisation Application (MAA).
The goal of EAMS is to give UK patients with life-threatening or significantly debilitating diseases early access to medications that have earned Promising Innovative Medicine (PIM) classification prior to marketing approval whenever there is a proven unmet medical need. Medicines approved for marketing by the MHRA and approved by the National Institute of Health and Care Excellence (NICE) are eligible for expedited NHS England commissioning.
The company applied to the MHRA for a United Kingdom Marketing Authorisation through the European Commission (EC) Decision Reliance Procedure after the Committee for Medicine Products for Human Use (CHMP) recommended approval of voxelotor in the European Union (EU). If authorized, voxelotor will be the first drug in the UK and Europe to target sickle hemoglobin polymerization, the underlying cause of sickling and red blood cell death in SCD.
SCD affects around 15,000 individuals in the UK
SCD is a rare hereditary disorder that affects about 15,000 individuals in the United Kingdom. Usually, SCD patients and their relatives go through a lifelong battle with the disorder that affects all parts of their lives. As a result, patients develop major problems and morbidities over time, including end-organ damage, resulting in a lower standard of living and early mortality. Additionally, many individuals with SCD have economic challenges and health inequalities, which can have detrimental societal consequences in areas like education, healthcare, and employment.
