Pfizer now has access to a premier sickle cell disease portfolio and pipeline, which has the potential to address pressing needs in a patient population that is underserved.
Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical business committed to the discovery, development, and delivery of game-changing therapies that give hope to underserved patient communities beginning with sickle cell disease, has been acquired by Pfizer Inc., it was announced today (SCD). The acquisition strengthens Pfizer’s dedication to SCD and builds on a 30-year history in the field of uncommon haematology.
GBT has a portfolio and pipeline that have the ability to meet all of this underserved community’s urgent requirements. GBT discovered and created Oxbryta® (voxelotor), a revolutionary drug that specifically addresses the underlying cause of SCD. Additionally, the U.S. Food and Drug Administration has designated GBT’s promising pipeline of preclinical and clinical investigational assets with a focus on SCD as an orphan drug and as having a rare paediatric disease, including GBT021601 (GBT601) and inclaclumab (FDA).
“With Global Blood Therapeutics’ talent, portfolio, and pipeline now part of Pfizer, we look forward to accelerating innovation and expeditiously bringing multiple potential best-in-class treatments to people living with sickle cell disease”, “In line with our value of equity, Pfizer is committed to addressing the underserved needs of the sickle cell disease community. We are excited about these potential breakthroughs and the opportunity to transform the lives of these patients.”
Aamir Malik, Chief Business Innovation Officer, Executive Vice President, Pfizer
Millions of people worldwide suffer from SCD, a fatal genetic blood illness that is most common in persons of African, Middle Eastern, and South Asian origin. Pfizer will maintain and expand on the businesses’ common dedication to and involvement in the SCD community.
Further Transaction Information
All of the outstanding shares of GBT’s common stock have been fully acquired by Pfizer at a price of $68.50 per share in cash, for a total estimated enterprise value of roughly $5.4 billion, including debt and less cash acquired. Today, Pfizer owns all of GBT’s subsidiaries. GBT’s common equity shares stopped trading on the NASDAQ Global Select Market as a result of the transaction.
Regarding Sickle Cell Disease
Hemolytic anaemia, acute pain crises, and progressive end organ damage are the hallmarks of the lifelong, disabling blood illness known as sickle cell disease (SCD). When sickled red blood cells irritate the blood vessel lining and trigger an inflammatory response that results in vascular occlusion, tissue ischemia, and pain, it is known as a vaso-occlusive crisis (VOC) or acute pain crisis. SCD complications start in childhood and are linked to a reduced life expectancy. Early diagnosis, management, and therapy of SCD have the ability to alter the course of the condition, lessen signs and symptoms, stop long-term organ damage, and increase life expectancy. Therapy that addresses the underlying aetiology of SCD and its acute and chronic consequences has historically had a high unmet demand. SCD is more common in persons with sub-Saharan African ancestry, while it can also affect those with Hispanic, South Asian, Southern European, and Middle Eastern ancestry.
Regarding Oxbryta® (voxelotor)
Patients with sickle cell disease can take Oxbryta (voxelotor) orally once day (SCD). Oxbryta functions by raising hemoglobin’s oxygen affinity. Since oxygenated sickle haemoglobin does not polymerize, Oxbryta prevents the sickling and red blood cell destruction that results in hemolysis and hemolytic anaemia, which are the main pathologies experienced by every person with SCD.
In December 2021, the FDA expanded the permitted uses of Oxbryta for the treatment of SCD in patients 4 years of age and older in the United States. The FDA granted expedited clearance for Oxbryta tablets in November 2019 for the treatment of SCD in adults and children 12 years of age and older. GBT will continue to research Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to evaluate the therapy’s capacity to reduce stroke risk in children 2 to 14 years of age, as a requirement of accelerated approval for patients ages 4 and older in the United States.
The FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for the treatment of patients with SCD in acknowledgment of the urgent need for novel SCD therapies. Oxbryta also won The Galien Foundation’s coveted Prix Galien USA prize for “Best Biotechnology Product” in 2021.
The European Medicines Agency (EMA) has given Oxbryta the Priority Medicines (PRIME) designation, the European Commission (EC) has designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD, and the Medicines and Healthcare products Regulatory Agency in the United Kingdom has given Oxbryta the Promising Innovative Medicine (PIM) designation (MHRA). The European Commission (EC) approved Oxbryta’s use as a monotherapy or in combination with hydroxycarbamide in February 2022 for the treatment of hemolytic anaemia caused by SCD in adult and paediatric patients 12 years of age and older (hydroxyurea). The treatment of hemolytic anaemia caused by SCD in adult and paediatric patients 12 years of age and older has been approved for marketing by the MHRA in Great Britain. Additionally, Kuwait, Oman, and the United Arab Emirates, which are members of the Gulf Cooperation Council, have granted Oxbryta marketing permission for the treatment of SCD in adults and children 12 years of age and older (UAE).
Important Safety Advice
If a patient has ever experienced an allergic reaction to voxelotor or any of the chemicals in Oxbryta, they shouldn’t take it. A list of Oxbryta’s ingredients can be found at the conclusion of the patient leaflet. Serious adverse events, particularly severe allergic ones, are possible with Oxbryta. Patients who experience a rash, hives, shortness of breath (difficult breathing), or swelling of the face should immediately inform their healthcare provider or seek emergency medical attention.
Headache, diarrhoea, stomach-area (abdominal) pain, nausea, rash or hives, and fever are among Oxbryta’s most frequent adverse effects. Children between the ages of 4 and under 12 are most likely to experience the following side effects with Oxbryta: fever, vomiting, rash, abdominal pain, diarrhoea, and headache. Not all of Oxbryta’s potential negative effects are listed here. Patients should inform their healthcare provider of all medical conditions before taking Oxbryta, including any liver issues, pregnancy plans (because it is unknown if Oxbryta can harm an unborn child), and breastfeeding (because it is unknown if Oxbryta can pass into breastmilk or if it can harm a baby). Patients should not breastfeed during treatment with Oxbryta and for at least 2 weeks after the last dose.
Patients should be honest about all of the medications they use with their doctor, including any prescription and over-the-counter drugs, vitamins, and herbal supplements. Some medications may alter how Oxbryta functions. Additionally, Oxbryta may alter the effectiveness of other medications as well as the outcomes of several blood tests.
For medical advice on side effects, patients are encouraged to call their doctor. You can contact the FDA to report side effects at 1-800-FDA-1088. You can also report side effects by calling 1-833-428-4968.
