Roche : FDA grants priority review for Roche’s Actemra/RoActemra (tocilizumab) supplemental biologics license application for giant cell arteritis, a form of vasculitis

Basel, 24 January 2017

FDA grants priority review for Roche's Actemra/RoActemra (tocilizumab) supplemental biologics license application for giant cell arteritis, a form of vasculitis

• Roche recently announced positive results from a Phase III clinical trial in giant cell arteritis (GCA)
• There have been no new therapies for GCA in more than 50 years
• Breakthrough therapy designation was granted to Actemra/RoActemra for the treatment of GCA by the FDA in 2016

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental biologics license application (sBLA) for Actemra/RoActemra (tocilizumab) for the treatment of GCA, a chronic, potentially life-threatening autoimmune condition. The FDA also granted priority review designation for Actemra/RoActemra for the treatment of GCA. The designation is based on the positive outcome of the Phase III GiACTA study evaluating Actemra/RoActemra in people with GCA. Results showed that Actemra/RoActemra, initially combined with a six-month steroid (glucocorticoid) regimen, more effectively sustained remission through one year compared to a six- or 12-month steroid taper regimen given alone in people with GCA.

'This positive outcome in GCA, a condition for which there have been no new treatments in more than 50 years, demonstrates Roche's commitment to helping patients with unmet needs,' said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. 'We are pleased by the FDA's decision to classify their review of the sBLA as priority. We will continue to work closely with the FDA to bring this investigational medicine to people with GCA as quickly as possible.'

Priority review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment of a serious disease.

In October 2016, Roche announced receiving breakthrough therapy designation for the treatment of GCA with Actemra/RoActemra. Breakthrough designation is intended to expedite the development and review of medicines with early evidence of substantial clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible.

About the GiACTA study

GiACTA (NCT01791153) is a Phase III, global, randomised, double-blind, placebo-controlled trial investigating the efficacy and safety of Actemra/RoActemra as a novel treatment for GCA. It is the largest clinical trial ever conducted in GCA and the first to use blinded, variable-dose, variable-duration steroid regimens. The multicentre study was conducted in 251 patients across 76 sites in 14 countries. The primary and key secondary endpoints were evaluated at 52 weeks.

About Giant Cell Arteritis

Giant cell arteritis (GCA) - also known as temporal arteritis (TA) - is a potentially life-threatening autoimmune condition. GCA has a global impact and usually affects those above the age of 50, and the disease is two-to-three-times more likely to affect women than men. GCA is often difficult to diagnose because of the wide and variable spectrum of signs and symptoms. GCA can cause severe headaches, jaw pain and visual symptoms and if untreated, can lead to blindness, aortic aneurysm or stroke. Treatment to date for people with GCA has been limited to high-dose steroids that play a role as an effective 'emergency' treatment option to prevent damage such as vision loss. However, steroids are often inadequate to maintain long-term disease control (flare-free remission). Due to the variability of symptoms, complexity of the disease and disease complications, people with GCA are often seen by several physicians including rheumatologists, neurologists and ophthalmologists.

About Actemra/RoActemra

Actemra/RoActemra is the only approved anti-IL-6 receptor biologic, available in both intravenous (IV) and subcutaneous (SC) formulations, for the treatment of adult patients with moderate to severe active rheumatoid arthritis (RA). Actemra/RoActemra can be used alone or with methotrexate (MTX) in adults who are intolerant to, or have failed to respond to other anti-rheumatic medications. In the most recent update to the European League Against Rheumatism (EULAR) RA management guidelines, Actemra/RoActemra is highlighted as the only biologic that has been repeatedly demonstrated to be superior as a monotherapy over MTX or other conventional disease-modifying antirheumatic drugs (DMARDs). The extensive Actemra/RoActemra RA IV clinical development program included five Phase III clinical studies and enrolled more than 4,000 people with RA in 41 countries. The Actemra/RoActemra RA SC clinical development program included two Phase III clinical studies and enrolled more than 1,800 people with RA in 33 countries. In Europe, Actemra/RoActemra IV and SC is also approved for use in adult patients with severe, active and progressive RA who previously have not been treated with MTX. Actemra/RoActemra IV formulation is approved in most major countries for polyarticular juvenile idiopathic arthritis (pJIA) and systemic juvenile idiopathic arthritis (sJIA) in children two years of age and older. Actemra/RoActemra is part of a co-development agreement with Chugai Pharmaceutical Co., Ltd and has been approved in Japan since April 2005. Actemra/RoActemra is approved in 115 countries worldwide.

Actemra/RoActemra is also being investigated in a global Phase III multicentre, randomised, double-blind, placebo-controlled study (NCT02453256) for patients with systemic sclerosis (SSc). Actemra/ RoActemra was granted breakthrough therapy designation for SSc by the US Food and Drug Administration (FDA) in June 2015.

About Roche in Immunology

The Roche Group's immunology medicines include: Actemra/RoActemra (tocilizumab) for rheumatoid arthritis and juvenile idiopathic arthritis; Rituxan/MabThera (rituximab) for rheumatoid arthritis granulomatosis with polyangiitis and microscopic polyangiitis; Xolair (omalizumab) in allergic asthma; Pulmozyme (dornase alfa) for cystic fibrosis; and Esbriet (pirfenidone) for idiopathic pulmonary fibrosis. Roche's immunology pipeline includes: RG7625, a cathepsin S antagonist; RG6125, a monoclonal antibody targeting cadherin-11; RG7845 (GDC-0853), a novel Bruton's tyrosine kinase (BTK) inhibitor; obinutuzumab for lupus nephritis and hypersensitised patients with end-stage renal disease; etrolizumab for ulcerative colitis and Crohn's disease; and lebrikizumab in a number of respiratory conditions and atopic dermatitis.

About Roche

Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people's lives.

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. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare - a strategy that aims to fit the right treatment to each patient in the best way possible.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. Twenty-nine 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. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry eight years in a row by the Dow Jones Sustainability Indices.

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2015 employed more than 91,700 people worldwide. In 2015, Roche invested CHF 9.3 billion in R&D and posted sales of CHF 48.1 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.