Despite decades of biomedical breakthroughs, 95% of the 7,000 identified rare diseases still lack an approved treatment or cure. While these diseases may be individually rare, they collectively impact 1 in 10 Americans. To address these unmet medical needs requires groundbreaking science in diseases that are not well understood. This high degree of complexity often means that mergers and acquisitions (M&A) are critically important for companies to be successful in bringing new treatments to patients.
Recent research underscores how important life sciences M&A can be for rare disease innovation. Drug projects in development by smaller, inexperienced companies that undergo M&A with a company that has prior experience launching a new drug are:
- Five times more likely to launch as novel drugs that fill an unmet medical need or represent an improvement over existing treatments,
- And nearly 24 times more likely to launch with a new mechanism of action, representing a treatment with an entirely new way of treating a disease.
Said differently, when smaller companies partner with experienced drugmakers through M&A, they unlock the capabilities and infrastructure needed to bring life-changing innovations to patients with significant unmet needs. Life sciences M&A has played a fundamental role in revolutionizing the treatment options available to patients with diseases such as:
- Pompe disease: The acquisition of Novazyme by Genzyme combined rare disease expertise and commercial/clinical experience to deliver Myozyme, the first-ever FDA-approved therapy for Pompe disease. This transformed a once-fatal disease into one where patients can live long and happy lives.
- Hypertrophic Cardiomyopathy: Bristol Myers Squibb’s acquisition of MyoKardia’s advanced mavacamtem, the first therapy to target the root cause of the disease, and the first new medicine approved to treat genetic cardiac diseases in over a decade.
- Myasthenia gravis: Johnson & Johnson’s (J&J) acquisition of Momenta brought IMAAVY to patients — leveraging Momenta’s specialized expertise and J&J’s regulatory and commercial infrastructure — offering new hope through a first-in-class therapy for patients with this debilitating autoimmune disease.
As these examples highlight, M&A is a unique and fundamental component of America’s world-class life sciences ecosystem, helping transform initial breakthroughs into life-changing therapies for patients with the greatest medical needs. For the 95% of rare diseases without an approved treatment, M&A could be the difference between a new treatment option or lack thereof. To sustain America’s leadership in life sciences innovation and the successes of the life sciences ecosystem, policymakers must recognize and support the unique role that pro-competitive M&A plays in advancing new treatments and cures.