Targeting the future of medicine

Our commitment to patients

At Blueprint Medicines, we work together in pursuit of one common goal: to improve and extend patients’ lives by solving important medical problems in allergy/inflammation and cancer.

We were founded on the belief that targeting the root cause of disease can dramatically improve the lives of patients. With our R&D and commercial expertise, we aim to rapidly identify important disease targets, design transformative medicines and deliver them to patients globally.

“Though deep scientific knowledge forms the basis of our company, we go beyond intellectual curiosity to achieve a common goal: to improve and extend the lives of patients
around the world.”
Fouad Namouni, M.D., President, Research & Development

Blueprint Medicines Recognizes International FOP Awareness Day

In honor of International FOP Awareness Day, our Chief Scientific Officer Marion Dorsch shares a letter to the patient community highlighting our commitment to advance care and raise awareness for those affected by this rare genetic disease.

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.

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Our approach

Our goal is to rapidly and reproducibly design medicines that treat the root cause of disease. With this approach, we aim to deliver important new medicines to patients in need.


core disease biology

Design a highly selective therapy

that targets the root cause of disease

Conduct clinical trials in patients

living with the disease

Diseases we target

Systemic mastocytosis

  • Systemic mastocytosis (SM) is characterized by the abnormal buildup of mast cells – immune cells that produce histamine and other mediators of the body’s inflammatory and allergic responses. In patients with SM, mast cells produce high levels of these mediators, causing symptoms that can range from mild to life-threatening, including pain, nausea, hives, bleeding, fever and anaphylaxis.

  • There are several forms of SM including indolent SM (ISM) and advanced SM. Many patients with ISM have a high symptom burden that severely impacts activities of daily living and quality of life. In advanced cases, including aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia, mast cells accumulate in organs such as the bone marrow, spleen and liver, often resulting in life-threatening organ dysfunction.

  • Until 2023, there had been no approved therapies for ISM that selectively target the KIT D816V mutation, the underlying driver of disease in approximately 95 percent of cases.

Hormone-receptor-positive, HER2-negative metastatic breast cancer and other CDK2-vulnerable cancers

  • CDK2 and Cyclin E (CCNE1) are central to regulating the cell cycle, which is involved in the process of cell growth and division.

  • CDK2 is believed to play an important role in tumor growth for patients with hormone-receptor-positive, HER2-negative breast cancer.

  • In subsets of patients with ovarian cancer and other tumor types, CCNE1 hyperactivates CDK2, resulting in tumor formation.

  • Targeting cancers vulnerable to CDK2 inhibition has the potential to deliver meaningful benefits for a broad range of patients.

Gastrointestinal stromal tumor

  • Gastrointestinal stromal tumor, or GIST, accounts for the most common sarcoma of the gastrointestinal tract, or GI tract. Tumors arise within cells in the GI tract wall, most often the stomach or small intestine.

  • Most patients are diagnosed between ages 50-80, typically triggered by GI bleeding, incidental findings during surgery or imaging, or in rare cases, tumor rupture or GI obstruction. Symptoms include pain or discomfort in the abdomen, nausea, vomiting, anemia and fatigue. Patients diagnosed at an early stage may undergo surgery.

  • Until 2020, there were no approved therapies for advanced GIST that selectively targeted PDGFRA exon 18 mutations, including PDGFRA D842V mutations.
Our clinical trials

Our clinical trials

Clinical trials are an important part of helping to bring safe and effective medicines to patients. All drugs go through rigorous series of trials, and based on the results, regulatory agencies including the U.S. Food and Drug Administration and European Medicines Agency decide whether to approve medicines.

By taking part in clinical trials, patients can make an important contribution to ongoing research and better understanding of a disease. For patients who lack effective treatments, clinical trials are also an opportunity to be a part of testing a new drug.

Learn more about participating in clinical trials
Explore our clinical trials

U.S. Patient Support Program

Designed to assist eligible patients who are prescribed our approved medicines by offering access and affordability solutions

Download Visit for more information

For questions, call 1-888-BLUPRNT (1-888-258-7768)
Monday-Friday, 8 a.m. to 8 p.m. Eastern Time (ET)