USA – Pfizer has completed its acquisition of Global Blood Therapeutics (GBT), a biopharma focused on drug discovery and development for rare blood disorders such as sickle cell disease (SCD) in a deal worth nearly US$5.4 billion.

The total enterprise value comprised debt and the net cash acquired. With the latest acquisition, GBT has become a wholly-owned Pfizer subsidiary.

Through the takeover, Pfizer will gain access to the portfolio and pipeline of GBT, which could possibly address the complete spectrum of vital needs for SCD patients.

This includes Oxbryta (voxelotor), a first-in-class medicine that directly targets the root cause of SCD and is approved in the United States for the treatment of SCD in patients aged four and up.

The drug is also approved in the European Union (EU), the United Arab Emirates (UAE), Oman, and the United Kingdom.

Oxbryta had 2021 net sales of US$195 million. Pfizer estimates that GBT’s pipeline and Oxbryta have the potential for a sickle-cell disease franchise that could achieve combined worldwide peak sales of more than US$3 billion.

SCD is a genetic disease that causes red blood cells to form a crescent shape, which can block blood vessels and affect how oxygen is transported throughout the body.

The disease can cause serious health problems such as anemia, fatigue, vaso-occlusive crises, and chronic end-organ damage.

GBT’s preclinical and clinical investigational assets for SCD also include GBT021601 (GBT601) and inclaclumab.

GBT021601 is a sickle hemoglobin (HbS) polymerization inhibitor, whereas inclacumab is a fully human monoclonal antibody that targets P-selectin.

P-selectin inhibition is clinically validated to reduce vaso-occlusive crises (VOCs) – a complication of SCD patients with frequent pain crises.

GBT has an exclusive worldwide licensing agreement with Roche for inclacumab, which has established pharmacokinetic data, safety and tolerability in more than 500 patients.

The FDA has granted these two assets Orphan Drug and Rare Pediatric Disease designations.

Other than Oxbryta and Inclaclumab, other emerging drugs for treating Vaso-occlusive-crisis-associated-with-sickle-cell-disease is intravenously administered L-citrulline developed by Asklepion Pharmaceuticals.

Intravenous L-citrulline, a non-essential amino acid is being developed by Asklepion, under a license from Vanderbilt University.

Despite recent advances in treatment, the majority of SCD patients continue to experience pain crises, lifelong disability, reduced quality of life, and shortened life expectancy, emphasizing the need for therapies that address the underlying cause of SCD as well as its acute and chronic complications.

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