BluePrint’s AYVAKIT and GAVRETO™ Selected Biologics and PantherRx Rare as Limited Specialty Pharmacies

Blueprint Medicines (Blueprint) is a global precision therapy company that invents life-changing therapies for people with cancer and hematologic disorders. Applying an approach that is both precise and agile, Blueprint creates medicines that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease.

Blueprint is delivering approved medicines directly to patients in the United States and Europe, and is globally advancing multiple programs for genomically defined cancers, systemic mastocytosis, and cancer immunotherapy. 

Blueprint has identified three key areas of strategic focus:

  • Advancing the company’s registrational programs and systemic mastocytosis.
  • Building its commercial expertise as the company has launched multiple new products.
  • Continuing to strengthen the pipeline with new investigational medicines from the company’s research platform.

Blueprint plans to accelerate global adoption of AYVAKIT and GAVRETO™ (pralsetinib):

  • AYVAKIT, a selective KIT and PDGFRA inhibitor, is approved in the U.S. and Europe for the treatment of patients with unresectable or metastatic gastrointestinal stromal tumor driven by certain PDGFRA mutations. The FDA recently approved Ayvakit (avapritinib) for the treatment of adults with unresectable (unable to be removed with surgery) or metastatic (when cancer cells spread to other parts of the body) gastrointestinal stromal tumor (GIST) – a type of tumor that occurs in the gastrointestinal tract, most commonly in the stomach or small intestine – harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. 
  • GAVRETO, a selective RET inhibitor, is approved in the U.S. for the treatment of patients with certain advanced or metastatic RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. 

Blueprint Medicines selected as limited distribution partners, PANTHERx® Rare Pharmacy  and Biologics by McKesson (Biologics), for both AYVAKIT and GAVRETO™. Both specialty pharmacies are leaders in dispensing orphan drugs for rare diseases.

Biologics offers more than 145 therapies, including more than 100 orals, at least 12 therapies exclusive to Biologics and 60 therapies in limited-distribution networks. As part of McKesson Life Sciences, Biologics’ services can be paired with a broader portfolio of solutions.

PANTHERx Rare is the nation’s leading rare disease pharmacy. PANTHERx was the first pharmacy nationwide awarded the Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC).

Also in 2020, Blueprint entered into a global collaboration with Roche and Genentech, a member of the Roche Group, to develop and commercialize pralsetinib. Under the collaboration, Blueprint Medicines and Genentech will co-commercialize pralsetinib in the U.S. and Roche will obtain exclusive commercialization rights for pralsetinib outside of the U.S., excluding Greater China. The companies also plan to expand development of pralsetinib in multiple treatment settings and explore development of a next-generation RET inhibitor as part of this collaboration.


Takeaway: Blueprint Medicines developing initiatives to accelerate global adoption of AYVAKIT and GAVRETO™ (pralsetinib)