Sometimes, the greatest things are discovered in the doing. As we have embarked on our mission to deliver life-changing medicines to people in need, we have brought together some of the sharpest minds to tackle the challenges of a rare, under-recognized disease called indolent systemic mastocytosis (ISM).
Today, we celebrate two years since our homegrown medicine AYVAKIT® (avapritinib) was approved for the treatment of ISM – and we’re fueled by the conviction that we’re just getting started.
A commitment to transforming the treatment landscape
One simple, yet ambitious belief has inspired us since the beginning: Patients deserve more than incremental improvements.
Starting with a deep understanding of human biology, our scientific approach is rooted in the design and development of potent, selective medicines targeting underlying drivers of disease. Early in our company’s history, we recognized the unmet patient needs that existed across the spectrum of systemic mastocytosis (SM). Our foundational research and development capabilities have enabled us to explore targets previously thought to be “undruggable,” including the underlying mutational driver of SM.
In collaboration with the broader medical and patient community, we put our science to the test by thoughtfully designing and conducting the largest clinical trial in ISM to-date1, subsequently achieving approvals from the U.S. FDA in May 2023 and the European Medicines Agency in December 2023.
The SM opportunity comes into focus
We always believed there were a significant number of people living with SM around the world, but emerging data showing an increase in diagnosed patients is helping us realize the prevalence is greater than previously thought and growing faster than we expected.2-3 To best serve this expanding population, our teams have redesigned how they partner across disciplines to accelerate education, awareness and access.
Today, awareness and clinical suspicion of SM is on the rise; with such a high medical need among patients, we believe healthcare providers are eager to pursue a timely diagnosis and appropriate treatment plan for their patients.
The grit to do more
For some, bringing forward one groundbreaking discovery to address a difficult-to-treat disease is the pinnacle. But for us at Blueprint Medicines, it’s our launchpad.
Over the past 15 years, we have honed our scientific and clinical knowledge of the critical role of the mast cell in mediating allergic and inflammatory responses. While targeting specific mediators after they are released has shown clinical benefit,4 our approach focuses on targeting the sources of disease, aiming to address factors contributing to disease development and progression rather than solely managing symptoms.
Today, we are applying our proven expertise in mast cell biology, track record of clinical execution and demonstrated commercial excellence to drive continued innovation within our SM franchise and across the universe of allergic and inflammatory disorders. We are doubling down on the promise of our science and our commitment to making a profound difference in people’s lives.