For most Americans, the term “mergers and acquisitions” (M&A) may not resonate beyond two companies coming together. In the case of biopharmaceutical M&A, the concept and the impact often mean a transformational medicine can make its way to patients far faster than one company going at it alone. For the hundreds of thousands of patients with sickle cell disease, a merger between like-minded companies helped make one such treatment a reality.
Sickle cell disease is an inherited blood disorder that alters red blood cells from the familiar red disc shape into the hard, sticky crescent form known as a “sickle.” These sickled cells are much more inflexible than typical blood cells, leading to blocked blood flow throughout the body. This blockage can lead to serious health problems for a patient: stroke, eye problems, infections, episodes of generalized pain and, worst of all, a significantly shorter life expectancy. Sickle cell disease is a lifelong illness; a bone marrow transplant is the only known cure.
Sickle cell disease is also what’s known as a rare disease, meaning it impacts fewer than 200,000 people in the U.S. There are currently more than 100,000 people living with sickle cell disease in the U.S., and 20 million people worldwide. Studies show that research for sickle cell disease, which predominately afflicts people of African descent, receives less funding and investment compared to other rare diseases, such as cystic fibrosis, which affects fewer than half the number of patients yet receives 3.5 times the funding from the National Institutes of Health and 440 times the funding from national foundations.
Observing the “terrible discrimination” his patients with sickle cell disease faced in his own medical practice is what inspired Ted Love, a Black physician who grew up in Alabama in the Jim Crow South, to co-found his company Global Blood Therapeutics. Upon seeing how “many medicines failed to benefit society’s most vulnerable,” Dr. Love recalled feeling he could “improve health care on a bigger scale through innovation” than he could as a practicing physician – and soon did. In 2019, the U.S. Food and Drug Administration (FDA) approved Global Blood Therapeutics’ novel sickle cell disease drug, the first oral therapy to treat the root cause of the disease. Seeking to achieve a wide distribution of this life-changing treatment, Love negotiated a deal to sell his company to Pfizer, aiming to take advantage of the company’s global reach and begin distribution to patients in Africa, India and South America.
Dr. Ted Love wrote for STAT+ recently about the value that pro-innovation M&A has provided not just to his company, but to individuals suffering from sickle cell disease across the world: “Mergers and partnerships are critical to ensure resources to conduct expensive clinical trials, secure regulatory approval, and then manufacture and distribute medicine at a national or global scale… This type of deal-making is particularly important for small companies. That was certainly the case at Global Blood Therapeutics. We could not have reached patients in lower-income countries, where child mortality from sickle cell disease is much greater, without being acquired…”
Dr. Love’s story of being inspired by his personal experiences with disease is not uncommon – consider the fact that sickle cell disease is just one of the 7,000 rare diseases known today, only five percent of which have an FDA-approved therapy. There are more than 2,300 biopharmaceutical companies in the U.S., with hundreds more starting every year in the hopes of bringing new treatments and cures to fight rare and other life-threatening diseases. Pro-innovation M&A policies, like those that allowed Global Blood Therapeutics to merge with a partner, make it possible for other companies pursuing these therapies to secure the resources, infrastructure and expertise needed to bring innovation from the lab to the patient.