Pfizer Acquires GBT in $5.4B Deal to Enhance Rare Hematology Presence

Global Blood Therapeutics was leader in SCD therapeutics

Mary Chapman avatar

by Mary Chapman |

Share this article:

Share article via email
Pfizer acquisitions | Sickle Cell Anemia News | handshake illustration

In a move to enhance three decades of treatment development in rare hematology, Pfizer will acquire Global Blood Therapeutics (GBT), a biopharmaceutical company that has been a leader in sickle cell disease (SCD) therapeutics, for $5.4 billion.

The acquisition brings GBT’s treatment portfolio and pipeline — and its sickle cell expertise — to Pfizer, with prospective total global sales of more than $3 billion. It potentially allows Pfizer to address the full spectrum of critical needs in SCD.

“Sickle cell disease is the most common inherited blood disorder, and it disproportionately affects people of African descent,” Albert Bourla, chairman and CEO of Pfizer , said in a press release.

“We are excited to welcome GBT colleagues into Pfizer and to work together to transform the lives of patients, as we have long sought to address the needs of this underserved community,” Bourla said.

Recommended Reading
orphan drug designation | Sickle Cell Disease News | announcement illustration of the word news

2 GBT Therapies Win FDA Orphan Drug, Rare Pediatric Designations

GBT brings extensive SCD portfolio

Pfizer plans to speed up the global distribution of Oxbryta (voxelotor), a first-in-class SCD treatment developed by GBT that directly targets the underlying mechanisms that cause sickle cell disease. The therapy works by increasing hemoglobin’s ability to bind oxygen, thus impeding clumping and red blood cell eradication.

Net sales of the oral therapy totaled approximately $195 million last year.

Great Britain granted Oxbryta marketing approval last month for the treatment of hemolytic anemia in SCD patients ages 12 and older. It previously had been approved in the U.K. for eligible patients under a governmental early access program.

The European Commission also approved the therapy earlier this year for the same patient population. Additionally, Oxbryta is available in the United Arab Emirates for those 12 and older.

The once-daily therapy was granted accelerated approval in the U.S. in 2019 for sickle cell patients ages 12 and older. That was extended last year to include children as young as 4.

“Today is an exciting milestone that accelerates GBT’s mission to discover, develop and deliver life-changing treatments that provide hope to underserved patient communities,” said Ted W. Love, MD, president and CEO of GBT.

“Pfizer will broaden and amplify our impact for patients and further propel much-needed innovation and resources for the care of people with sickle cell disease and other rare diseases, including populations in limited-resource countries,” Love said, adding, “We look forward to working together with Pfizer to serve our communities and advance our shared goal of improving health equity and expanding access to life-changing treatments to create a healthier future for all.”

In other SCD news, GBT has launched a new trial assessing the safety, tolerability, effectiveness, and pharmacological properties of its oral therapy GBT601. Initial data from the Phase 2/3 clinical trial (NCT05431088) are expected by year’s end.

This experimental treatment is considered the next-generation version of Oxbryta. It is based on the same mechanism of action, but is expected to have a more potent effect when given at lower doses.

GBT also is developing inclacumab, an antibody designed to reduce the risk of painful SCD-related attacks. Such attacks, which result from blood vessel blockage, are known as vaso-occlusive crises (VOCs).

Its efficacy and safety are being evaluated in two Phase 3 trials. One of the studies, called GBT2104-131 (NCT04935879), is testing inclacumab’s ability to lower the risk of VOCs. The second trial, known as GBT2104-132 (NCT04927247), is assessing whether a single dose of the therapy can reduce the risk of future hospitalization re-admissions due to these crises.

Both inclacumab and GBT601 have been granted both orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration.

“The deep market knowledge and scientific and clinical capabilities we have built over three decades in rare hematology will enable us to accelerate innovation for the sickle cell disease community and bring these treatments to patients as quickly as possible,” Bourla said.