Mustang Bio, Inc. (MBIO) and St. Jude Children’s Research Hospital have announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA).
Each year, thousands of drugs and devices move through the Food and Drug Administration’s regulatory pathways. In July alone, the agency approved some 400 drugs. And the FDA isn’t just looking at medications that are traditionally thought of as treatment—it’s also monitoring novel devices, smartphone apps and cell therapies, to name a few. These therapies are opening up new ways to think about treatment, balancing concerns like clinical outcomes, patient experience, risky side effects and mounting costs. That, in turn, has the potential to disrupt people’s understanding of the pharmaceutical industry more broadly.
In less than two months since late June, four digital health companies—Health Catalyst, Livongo Health, Phreesia and Change Healthcare—have gone public. At least one more, Peloton, might launch its own initial public offering before the year is out. Nashville, Tenn.-based Change Healthcare is making its own push to increase efficiencies in the healthcare market with software and analytics products that target several areas including a company’s overall financial performance, payment accuracy and financial, administrative and clinical transactions.