Letter from Marion Dorsch, Chief Scientific Officer
Today, Blueprint Medicines stands with the patient community and recognizes International Fibrodysplasia Ossificans Progressiva (FOP) Awareness Day. In doing so, we hope to draw attention to this rare genetic disease and the importance of advancing care for those affected.
At Blueprint Medicines, many of our scientists have worked on designing our experimental medicine over the past four years—what we call BLU-782. This is emblematic of our commitment to help the FOP patient community, who needs safe and effective treatment options. We recognize this need and are urgently trying to bring new medicines to patients.
One of the key reasons I was motivated to join Blueprint Medicines was the company’s FOP research. I learned about FOP earlier on in my career, and because the disease has a clearly defined cause, I thought there was the potential to overcome it with a precisely targeted treatment. Our company’s focus is targeting the underlying cause of diseases with precision medicines, which have the potential to make a transformative difference for patients. Our experimental medicine for FOP is a hallmark of this approach.
Currently, we are in the first stage of human testing to evaluate the safety of our experimental therapy. Based on the progress of the ongoing Phase 1 clinical trial in healthy volunteers, and input from clinical experts and the patient community, we have recently announced plans to initiate a Phase 2a clinical trial in patients with FOP in the fourth quarter of 2019.
We look forward to hearing from the patient community to learn more about how FOP affects the lives of people living with it, what is important to them and what a new treatment should offer, how we can optimize the design and conduct of our clinical trials, and new ways we can partner with the community on broader initiatives that can advance treatment, overall care and outcomes.
Those of us who work here at Blueprint Medicines do so in the pursuit of one common goal: to dramatically improve the lives of people with rare diseases. We want to thank those in the patient community, including the International FOP Association, for their leadership and unwavering support of people living with FOP worldwide. We are honored to work with you as we collectively make strides toward our ultimate goal of improving patients’ lives. No person with FOP should embark on this journey alone—and we at Blueprint Medicines will continue to join with advocates, clinicians and others to stand by you, side by side.