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Roche announces FDA approval of Gavreto (pralsetinib) for people with advanced and metastatic RET-mutant positive fusion thyroid cancers and RET positive fusion


Basel, 2 December 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the US Food and Drug Administration (FDA) has approved Gavreto ™ (pralsetinib) for the treatment of adult and pediatric patients 12 age and older with advanced or metastatic rearrangement during transformation (RET) – a common benign thyroid cancer (MTC) that requires systemic therapy, or with advanced or metastatic RET positive fusion thyroid cancer that requires systemic therapy and is radioactive iodine refractory (if radioactive iodine is appropriate). These indications were approved under the FDA’s accelerated approval program based on data from the ARROW phase I / II study. Ongoing approval for these indications may be subject to validation and description of clinical benefit in confirmatory trials.

“We are pleased to partner with Blueprint Medicines to bring this important new option to people with some types of RET-modified thyroid cancer,” said Levi Garraway, MD, Ph.D., Roche Chief Medical Officer and Head of Global Product Development. “Gavreto has now been approved across several types of tumors that have been changed by RET, underlining our commitment to promoting personalized healthcare with treatments that target the basic cancer biology of each individual.”

About 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer)1 having RET positive fusion tumors,2 and about 90% of people with advanced MTC (a rare form of thyroid cancer) carry RET mutations.3 Biomarker testing for RET fusion and mutations can help identify people eligible for treatment with Gavreto.

The approvals are based on the results of the ARROW phase I / II study, which demonstrated durable clinical activity in people with or without prior therapy and irrespective of RET switch genotypes.4 Treatment with Gavreto resulted in an overall response rate (ORR) of 60% (95% CI: 46%, 73%) in 55 people with RET-mutant metastatic MTC previously treated with cabozantinib and / or bandetanib, and duration the median of the response. Not reached (DoR) (95% CI: 15.1 months, not estimable).2 In 29 people with advanced RET-mutant cabozantinib- and vandetanib-naïve MTC who were determined not to be eligible for standard therapies, the ORR was 66% (95% CI: 46%, 82%), and the median DoR was not reached. (95% CI: not estimable, not estimable).4 In nine people with metastatic thyroid cancer RET positive fusion, Gavreto showed an ORR of 89% (95% CI: 52%, 100%), and the median DoR was not reached (95% CI: not estimable, it is not ‘ n estimable).4 In ARROW trial patients across RET-modified tumor types, the most common adverse reactions (≥25%) were constipation, increased blood pressure (hypertension), fatigue, musculoskeletal pain and diarrhea.4

The FDA reviewed and approved the application under its Real Time Oncology Review (RTOR) pilot program, which seeks to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible. In September, the FDA granted Gavreto accelerated approval for the treatment of adults with RET metastatic fusion-cell lung cancer (NSCLC) as found by an FDA-approved test. Ongoing approval for this indication may be subject to validation and description of clinical benefit in a confirmatory trial. In addition, the FDA granted Gavreto a New Breakthrough Therapy Designation for the treatment of RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments and for RSC positive fusion NSCLC that have progressed following chemotherapy platinum.

Gavreto is a once-a-day oral precision therapy designed to selectively target RET adjustments, including fusion and mutations.

About the ARROW study5
ARROW (NCT03037385) is a first-person, open-label, phase I / II study designed to evaluate the safety, tolerance and efficacy of Gavreto, orally administered in non-small positive fusion transfusion (RET) transfusion . cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC), RET fusion-positive thyroid cancer and other RET-modified solid tumors. The trial consists of two parts: a dose escalation portion, which is complete, and an expanding dose in people treated with 400 mg of Gavreto, once daily. ARROW is held at several sites across the United States, the European Union and Asia.

About cancers changed by RET
RET gene modifications, such as fusion and mutations, are key disease drivers in many types of cancer, including NSCLC and many types of thyroid cancer. About 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer)1 having RET positive fusion tumors,2 and about 90% of people with advanced MTC (a rare form of thyroid cancer) carry RET mutations.3 In NSCLC, RET fusion represents approximately 1-2% of patients.6 Oncogenic RET fusion is also seen at low frequencies in cholangiocarcinoma, colorectal, neuroendocrine, ovarian, pancreatic and thymus cancers.

About Gavreto
Gavreto is a once-daily oral precision therapy designed to selectively target RET modifications, including fusion and mutations, regardless of tissue of origin. Preclinical data has shown that Gavreto prevents primary RET fusion and cancer-causing mutations in subsets of patients, as well as secondary RET mutations that are predicted to drive resistance to treatment. Blueprint Medicines and Roche are jointly developing Gavreto for the treatment of patients with different types of RET-modified cancers.

Blueprint Medicines and Roche co-develop Gavreto globally, with the exception of Greater China. * Blueprint Medicines and Genentech, a wholly owned member of Roche Group, will co-commercialize Gavreto in the US and Roche has exclusive commercialization rights to Gavreto outside the US. , except Greater China. *

The Roche
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche a leader in personalized healthcare – a strategy that seeks to best fit the right treatment for each patient.

Roche is the world’s largest biotechnology company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and central nervous system diseases. Roche is also a world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a leader in diabetes management.

Founded in 1896, Roche continues to seek better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical devices by working with all relevant stakeholders. More than thirty medicines developed by Roche have been included in the World Health Organization Model Lists of Essential Medicines, and include antibiotics, antivirals and life-saving cancer medicines. In addition, for the twelfth year in a row, Roche has been recognized as one of the most sustainable companies in the Pharmaceutical Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, operates in over 100 countries and in 2019 employed around 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 billion. Genentech, in the United States, is a wholly owned member of Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, visit

* Greater China covers Mainland China, Hong Kong, Macau and Taiwan

All trademarks used or mentioned in this statement are protected by law.

[1] American Cancer Treatment Centers. Types of Thyroid Cancer [Internet; cited 2020 October]. Available from:
[2] Santoro M et al. RET Gene Fuses in Thyroid Malignancies and Other Tissues. Genes 2020; 11 (4): 424
[3] Li A et al. RET fusion in solid tumors. Cancer Treatment Rev. 2019; 81: 101911
[4] Blueprint Remedies. Data on file.
[5] Phase 1/2 Study of the highly selective RET Inhibitor, Pralsetinib (BLU-667), in Patients with Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors (ARROW) [Internet; cited 2020 October]. Available from:
[6] Drilon et al. Targeting RET-driven Cancers: Lessons from Evolving Landscape and Clinical Impacts. Nat Rev Oncol Clinic. 2018; 15: 151‒67.

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