Pfizer Fully Acquires Global Blood Therapeutics, Oxbryta for SCD

$5.4B acquisition includes inclacumab, potential therapy for VOCs in Phase 3 trials

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by Mary Chapman |

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In a $5.4 billion deal, Pfizer fully acquired Global Blood Therapeutics (GBT), bolstering its decades-long standing in the field of rare blood disorders, particularly regarding sickle cell disease (SCD).

With the newly completed acquisition — plans were announced in August — Pfizer gains GBT’s therapy portfolio and pipeline. The biopharmaceutical company also stands to benefit from GBT’s expertise in sickle cell, potentially permitting it to act on the broad range of the community’s crucial needs.

Pfizer, a 30-year leader in its own right in the space of rare blood disorders, has pledged to continue building upon that legacy and the companies’ mutual commitment to, and engagement with, SCD patients, families, and other stakeholders.

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Most people with the inherited disorder that affects red blood cells are of African, Middle Eastern, and South Asian descent. Of the 120 million people around the world thought to have SCD, more than 66% live in Africa. In the U.S., 1 in 13 African Americans have the sickle cell trait, meaning their children are at risk of the disorder.

“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,” Aamir Malik, chief business innovation officer and executive vice president at Pfizer, said in a company press release.

Oxbryta added to Pfizer’s portfolio of blood disorder therapies

“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,” Malik added.

Among the company’s planned steps is advancing worldwide distribution of Oxbryta (voxelotor), an oral SCD therapy developed by GBT that targets the disease’s root cause. The treatment works by increasing hemoglobin’s ability to bind oxygen, helping to prevent clumping and red blood cell elimination. Hemoglobin is the protein in red blood cells that is responsible for oxygen transport.

Oxbryta was approved in the U.S. in 2019 for SCD patients ages 12 and older, and its use was extended to children as young as 4 in December. It also gained marketing approval in the U.K. for people ages 12 and older in July, after being made available for qualified patients there under an early access program.

The therapy has also been approved across the European Union to treat SCD patients ages 12 and older, when used alone or in combination with hydroxyurea. Pfizer reports that Oxbryta is also available in Kuwait, Oman, and the United Arab Emirates for patients ages 12 and older.

In addition to Oxbryta, Pfizer acquired GBT’s pipeline of investigational SCD therapies, including GBT601 and inclacumab, both of which were given orphan drug and rare pediatric disease designations in the U.S.

Two ongoing Phase 3 trials are investigating inclacumab, an intravenous therapy designed to reduce the risk of vaso-occlusive crises (VOCs), the painful SCD-related attacks resulting from sickled cells blocking blood vessels. One, called GBT2104-131 (NCT04935879), is enrolling up to 240 patients, ages 12 and older, at sites worldwide, including across the U.S., Europe, South America, and Africa. It is testing whether inclacumab taken every 12 weeks can reduce the risk of VOCs relative to a placebo.

GBT2104-132 (NCT04927247), the other Phase 3 study, is testing the ability of a single dose of inclacumab, against a placebo, to lower the risk of future hospital re-admissions due to VOCs. This global trial  is now enrolling up to 280 patients, also starting at age 12, who had an index VOC requiring hospital care.