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Blueprint Medicines Announces FDA Approval of GAVRETO™ (pralsetinib) for the Treatment of Patients with Advanced or Metastatic RET-Mutant & RET Fusion-Positive Thyroid Cancer

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A precision therapy company focused on genomically defined cancers, rare diseases, and cancer immunotherapy, today announced that the U.S. Food and Drug Administration (FDA) has approved GAVRETO™ (pralsetinib) for the treatment of patients with RET-altered thyroid cancers. The accelerated approval expands the labeled indications for GAVRETO to include adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). Developed by Blueprint Medicines, GAVRETO is a once-daily oral precision therapy designed to potently and selectively target RET alterations that drive multiple tumor types. GAVRETO is jointly commercialized in the U.S. by Blueprint Medicines and Genentech, a wholly owned member of the Roche Group, under Blueprint Medicines' collaboration with Roche.

In the Phase 1/2 ARROW trial, GAVRETO showed durable efficacy and was generally well-tolerated in patients with RET-altered thyroid cancers with or without prior systemic therapy. Earlier this year, the FDA granted accelerated approval to GAVRETO for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test.

"With this approval, Blueprint Medicines has achieved four marketing authorizations this year across our lead programs, making real our vision to bring transformative precision therapies to patients globally," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Today's approval also builds further momentum toward bringing GAVRETO to a wide range of patients with RET-altered cancers. Now, as we work with our partner Genentech to rapidly deliver GAVRETO to patients with RET-altered non-small cell lung cancer and thyroid cancers, we continue to explore the potential of GAVRETO to address additional tumor types and treatment settings."

"Traditionally, we have treated patients with RET-altered thyroid cancers with multi-kinase inhibitors, non-selective therapies with modest efficacy, and clinically significant side effects. The FDA approval of pralsetinib (GAVRETO), a once-daily RET-targeted therapy, advances the standard of care for these patients," said Mimi Hu, M.D., professor in the Department of Endocrine Neoplasia and Hormonal Disorders at The University of Texas MD Anderson Cancer Center, and an investigator on the ARROW trial. "As a clinical researcher with a focus on thyroid cancer, I am encouraged by the safety profile and durable responses shown by GAVRETO in RET-altered thyroid cancers in both treatment-naïve and previously treated patients."

This approval is based on efficacy and safety results from the ARROW trial.1 In 55 patients with RET-mutant MTC previously treated with cabozantinib or vandetanib, the overall response rate (ORR) was 60 percent (95% CI: 46%, 73%), and the median duration of response (DOR) was not reached (95% CI: 15.1 months, not estimable). In 29 cabozantinib and vandetanib-naïve patients with RET-mutant MTC who were not candidates for standard systemic therapy per the study protocol, the ORR was 66 percent (95% CI: 46%, 82%), and the median DOR was not reached (95% CI: not estimable, not estimable). In addition, the ORR was 89 percent (95% CI: 52%, 100%) in nine patients with RET fusion-positive thyroid cancer, and the median DOR was not reached (95% CI: not estimable, not estimable). In ARROW trial patients across RET-altered tumor types, the most common adverse reactions (≥25%) were constipation, hypertension, fatigue, musculoskeletal pain, and diarrhea.

The continued approval of GAVRETO for advanced or metastatic RET-altered thyroid cancers may be contingent upon verification and description of clinical benefit in confirmatory trials. The FDA approved these additional indications for GAVRETO under its Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

"The FDA approval of GAVRETO has the potential to address important medical needs for patients with RET-altered thyroid cancers, and reflects a shift in care toward precision medicines designed to target the underlying driver of disease," said Gary Bloom, executive director of ThyCa: Thyroid Cancer Survivors' Association ( "We are excited that the emergence of targeted therapies like GAVRETO, combined with the expanded use of biomarker testing, may dramatically improve treatment for the RET-altered thyroid cancer community. At ThyCa, we are committed to advancing awareness of these promising areas of clinical research, educating our members on the importance of biomarker testing and offering support for those impacted by the disease."

Biomarker testing for RET enables clinicians to identify patients who are candidates for treatment with GAVRETO. RET alterations can be identified with available biomarker tests, including next-generation sequencing with tumor tissue or liquid biopsies.

About GAVRETO (pralsetinib)

GAVRETO (pralsetinib) is a once-daily oral targeted therapy approved by the FDA for the treatment of three indications: adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant MTC who require systemic therapy, and adults and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).

GAVRETO is not approved for the treatment of any other indication in the U.S. by the FDA or for any indication in any other jurisdiction by any other health authority.

GAVRETO is designed to selectively and potently target oncogenic RET alterations, including secondary RET mutations predicted to drive resistance to treatment. In pre-clinical studies, GAVRETO inhibited RET at lower concentrations than other pharmacologically relevant kinases, including VEGFR2, FGFR2, and JAK2.

Blueprint Medicines and Roche are co-developing GAVRETO globally (excluding Greater China) for the treatment of patients with RET-altered NSCLC, various types of thyroid cancer, and other solid tumors. The European Medicines Agency validated a marketing authorization application for GAVRETO for the treatment of RET fusion-positive NSCLC. The FDA granted breakthrough therapy designation to GAVRETO for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of GAVRETO in Greater China, which encompasses Mainland China, Hong KongMacau, and Taiwan.

About RET-Altered Solid Tumors

RET activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and multiple types of thyroid cancer. RET fusions are implicated in approximately 1 to 2 percent of patients with NSCLC and approximately 10 to 20 percent of patients with papillary thyroid cancer, while RET mutations are implicated in approximately 90 percent of patients with advanced MTC. In addition, oncogenic RET fusions are observed at low frequencies in colorectal, breast, pancreatic, and other cancers, as well as in patients with treatment-resistant EGFR-mutant NSCLC.

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Programs 9/23/21