A change of paradigm for sickle cell disease?

Recent months have seen two important acquisitions in the biopharma sector relating to sickle cell disease (SCD). In August, the pharmaceutical giant Pfizer purchased Global Blood Therapeutics, Inc. (GBT) for $5.4 billion. GBT is a biopharmaceutical company notably known for the development of a new drug used to treat sickle cell patients by inhibiting hemoglobin S (HbS) polymerization, and for having a portfolio and pipeline focused on SCD. Pfizer's stated positioning is to accelerate innovation to rapidly bring new treatments to sickle cell patients.

In October, Novo Nordisk acquired Forma Therapeutics for $1.1 billion. Forma Therapeutics has developed a selective pyruvate kinase-R activator to treat rare genetic blood disorders such as SCD. This drug is a once-daily oral medication that has been given rare pediatric disease and orphan drug status by the FDA and is currently undergoing a phase 2/3 study. This latest takeover is in line with the goal of Novo Nordisk to advance its scientific presence and pipeline in hemoglobinopathies.

The question is what impact these acquisitions could have on SCD stakeholders? These deals are historical. This is a clear statement that SCD is of interest to big players in the healthcare sector. There will be more money injected into clinical development and this should accelerate access to new and effective treatments for sickle cell patients, addressing a major medical need in the sickle cell field.

It is too early to see the consequences of these acquisitions in the field of SCD, but we expect it could be an opportunity for a real change of paradigm for sickle cell patients.