• Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations
• The approval was granted only 4 months (126 days) after submission of the dossier
  

Basel, 15 October 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today has announced that the Swiss agency for therapeutic products (Swissmedic) has granted a temporary approval for Gavreto^(R) (pralsetinib) in the following indications:¹

• RET-fusion positive Non-Small Cell Lung Cancer: Gavreto is indicated for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) who require systemic therapy and who have experienced progression after prior treatment.
• RET-mutant Medullary Thyroid Cancer: Gavreto is indicated for the treatment of adult patients with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy and who have experienced progression after prior treatment with tyrosine kinase inhibitors.
• RET-fusion positive Thyroid Cancer: Gavreto is indicated for the treatment of adult patients with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who have experienced progression after prior treatment including radioactive iodine.
  
  

These indications were approved based on data from the global phase I/II ARROW study.²

“We are proud that this approval occurred only 4 months (126 days) after submission of the dossier, which represents one of the fastest approvals of a new active substance (NAS) in Switzerland”, said Corinne Wenger, Head of Regulatory Affairs at Roche Pharma Switzerland. “Bringing this new treatment option for multiple RET-altered tumour types in such a fast timespan to the Swiss patients is the result of an efficient collaboration between Roche and the agency.”

The revised Therapeutic Product Act (TPA), article 9 and 13, allow accelerated timelines due to designation of a temporary authorisation status and in addition Orphan Medical Products with NAS the reliance on a decision of a reference authority (in this case the FDA). The temporary authorisation is contingent on the timely fulfilment of conditions.¹ After they have been met, the temporary authorisation can be transformed into an ordinary authorisation.

RET-activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and medullary thyroid cancer (MTC), and treatment options that selectively target these genetic alterations are limited.³ Biomarker testing for these fusions is the most effective way to identify people who are eligible for treatment with Gavreto.^1,3

Gavreto is now the sixth Swissmedic-approved medicine in Roche’s portfolio of treatments for lung cancer and Roche’s first approved targeted therapy for thyroid cancer. Gavreto will be available for Swiss patients by the end of October 2021.

About the ARROW study²
ARROW (NCT03037385) is a phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC), RET fusion-positive thyroid cancer and other RET-altered solid tumours. The trial consists of two parts: a dose escalation portion, which is complete, and an expansion portion in people treated with 400 mg of Gavreto, once-daily. ARROW is being conducted at multiple sites across the United States, European Union and Asia.^2,5,6

In the ARROW study Treatment with Gavreto led to an overall response rate (ORR) of 61% (95% CI: 50%, 71%) in 87 patients with NSCLC and previous platinum-based chemotherapy, including five (6%) patients with a complete response.1,5 In 55 people with RET-mutant metastatic MTC, previously treated with cabozantinib and/or vandetanib, treatment with Gavreto led to an overall response rate (ORR) of 60% (95% CI: 46%, 73%).6 In nine people with RET fusion-positive metastatic thyroid cancer, Gavreto demonstrated an ORR of 89% (95% CI: 52%, 100%).⁶ The safety of Gavreto was evaluated in 471 patients treated with 400 mg, once daily (QD) in the ARROW trial. The most common serious adverse drug reactions (ADR) were pneumonia (10.0%), pneumonitis (5.1%), anemia (3.0%) and urinary tract infection (3.0%).^1,5,6

About RET-altered cancers
RET gene alterations, such as fusions and mutations, are key disease drivers in many types of cancer, including NSCLC and several types of thyroid cancers. Approximately 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer) have RET fusion-positive tumours, and roughly 90% of people with advanced MTC (a rare form of thyroid cancer) carry RET mutations.^3,4 In NSCLC, RET fusions represent approximately 1-2% of patients.3 Oncogenic RET fusions also are observed at low frequencies in cholangiocarcinoma, colorectal, neuroendocrine, ovarian, pancreatic and thymus cancers.³

About Gavreto(R) (pralsetinib)
Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment.
Blueprint Medicines and Roche are co-developing Gavreto for the treatment of people with various types of RET-altered cancers.

About 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, as well as growing capabilities in the area of data-driven medical insights help Roche deliver truly personalised healthcare. Roche is working with partners across the healthcare sector to provide the best care for each person.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. In recent years, Roche has invested in genomic profiling and real-world data partnerships and has become an industry-leading partner for medical insights.

Founded in 1896, Roche continues to search for 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 innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com.

All trademarks used or mentioned in this release are protected by law.
Blueprint Medicines, Gavreto and associated logos are trademarks of Blueprint Medicines Corporation.

References
[1] Swissmedic Fachinformation Gavreto, www.swissmedicinfo.ch
[2] ClinicalTrials.gov. 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: https://clinicaltrials.gov/ct2/show/NCT03037385
[3] Drilon et al. Targeting RET-driven cancers: lessons from evolving preclinical and clinical landscapes. Nat Rev Clin Oncol. 2018;15:151‒67.
[4] Salvatore et al.The importance of the RET gene in thyroid cancer and therapeutic implications. Nat Rev Endocrinol. 2021 May;17(5):296-306.
[5] Gainor et al. Pralsetinib for RET fusion-positive non-small-cell lung cancer (ARROW): a multi-cohort, open-label, phase 1/2 study. Lancet Oncol. 2021 Jul;22(7):959-969.
[6] Subbiah et al. Pralsetinib for patients with advanced or metastatic RET-altered thyroid cancer (ARROW): a multi-cohort, open-label, registrational, phase 1/2 study. Lancet Diabetes Endocrinol. 2021 Aug;9(8):491-501.

Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche.com

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