Innovations Advanced by M&A: The First Therapy for Pompe Disease

May 1, 2024 | Blog Post, Innovations Advanced by M&A

This blog is another installment in our series, Innovations Advanced by M&A, which underscores the importance of mergers and acquisitions in ensuring a competitive and vibrant biopharmaceutical landscape for patients. You can find previous blogs in this series here:

Traversing the path to bring a breakthrough medicine to patients is a unique, high-risk endeavor that can take over a decade. From the complex scientific and regulatory expertise required, to the enormous capital investment needed for state-of-the-art facilities and clinical trials, many companies cannot navigate this path alone. Mergers and acquisitions (M&A) are fundamental to shepherding medicines along the path to market, and to patients in need. Such was the case for a first-of-its-kind treatment for Pompe disease.

Pompe disease is a rare genetic disease that causes progressive muscle deterioration, with most patients passing away before their first birthday. Pompe disease was first identified in 1932 by Dutch neurologist Dr. Johannes Pompe, but it wasn’t until 2006 that the first treatment for Pompe was brought to market. M&A helped make this long-anticipated breakthrough possible.

The Role of M&A:

In 1998, John and Aileen Crowley’s lives were changed forever when their 15-month-old daughter and newborn son were both diagnosed with Pompe disease. Acting quickly to save the lives of his children, John partnered with Pompe disease researcher Dr. William Canfield to found Novazyme, a small biotech based in Oklahoma City dedicated to bringing a treatment to patients with Pompe.

“We don’t consider any company to be the competition. The competition is the disease.”

John Crowley, Novazyme co-founder (2001)

As Novazyme exhausted its initial start-up funding, the company sought additional investment and resources. In 2001, Novazyme was acquired by Genzyme, a commercial-stage company with extensive experience in genetic disorders – including Pompe disease. Through the combination of Novazyme’s specialized focus on Pompe disease with Genzyme’s clinical and commercial experience, the combined company could devote unprecedented expertise and resources to bring a Pompe therapy to market.

“Novazyme and Genzyme have a shared vision of bringing the very best medicine to very sick patients as quickly as possible. Genzyme’s infrastructure, resources, and extensive experience in the area of genetic disorders can powerfully support and accelerate the development of our technology and products. This merger is an important event for patients… and further advances our mission.”

John Crowley, Novazyme (2001)

In 2006, under the direction of John and Dr. Canfield, the combined company found long-awaited success in Myozyme, the first-ever FDA-approved therapy for Pompe disease. Over twenty years after the diagnoses that changed their lives, John and Aileen’s daughter and son, who both received Myozyme, recently celebrated their 26th and 25th birthdays.

“[Genzyme] helped to develop what became the first generation therapy that three years later the kids received, that they’ve been receiving now for 13 years.”

John Crowley, Novazyme co-founder (2016)

The therapy that helped save the lives of the Crowley children, and others, illustrates the unique and fundamental role of M&A in supporting life sciences innovation, and how that innovation can often translate to life-changing therapies. With 10,000 known rare diseases, and only a few hundred with effective treatments available, pro-innovation M&A is critical to help support the next generation of biopharmaceutical breakthroughs for patients.