Roche : CHMP recommends EU approval for subcutaneous formulation of RoActemra for use in active systemic juvenile idiopathic arthritis (sJIA), a rare form of juvenile arthritis

Basel, 21 September 2018

CHMP recommends EU approval for subcutaneous formulation of RoActemra for use in active systemic juvenile idiopathic arthritis (sJIA), a rare form of juvenile arthritis

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the subcutaneous (SC) formulation of RoActemra® (tocilizumab) for the treatment of active systemic juvenile idiopathic arthritis (sJIA) in patients one year of age and older. RoActemra can be given alone or in combination with methotrexate (MTX) in patients with sJIA. In 2011, the European Commission approved the intravenous (IV) formulation of RoActemra for patients two years of age and older with active sJIA.

'Although rare, systemic juvenile idiopathic arthritis is a debilitating disease affecting children early in their lives,' said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. 'Today's announcement offers hope for more flexibility to these young patients, who could soon be prescribed either RoActemra IV, administered in a medical office, or RoActemra subcutaneous, a prefilled syringe that can be injected at home.'

sJIA is the rarest form of juvenile idiopathic arthritis (JIA), a chronic arthritic disease affecting children.¹ sJIA is characterised by inflammation in one or more joints, and a daily, spiking fever for at least two weeks, which may be accompanied by a skin rash.¹ Other symptoms may include anaemia, enlargement of the liver or spleen, and inflammation of the lining of the heart and/or lungs.¹

The positive CHMP opinion is based on data from the WA28118 study, a 52-week, open-label, multicentre, phase Ib pharmacokinetic (PK)/pharmacodynamic (PD) bridging study designed to determine the appropriate dosing regimen of RoActemra SC across a range of body weights (BWs) in children with sJIA.² The study enrolled 51 patients aged one to 17 years with sJIA and inadequate response to nonsteroidal anti-inflammatory drugs (NSAIDs) and corticosteroids who were either RoActemra naive or were receiving RoActemra IV with adequate disease control. RoActemra SC was administered open label according to a BW-based dosing regimen: sJIA patients weighing <30 kg received 162 mg of RoActemra every two weeks or 10 days and sJIA patients weighing ≥30 kg received 162 mg of RoActemra every week for 52 weeks. Model-computed PK and PD parameters, and safety were assessed.

In general, the safety observed for RoActemra SC was consistent with the known safety profile of RoActemra IV, with the exception of injection site reactions (ISRs). A higher frequency of RoActemra SC patients experienced ISRs, 41% (21/51) compared to patients treated with RoActemra SC for other approved indications. All ISRs reported were non-serious, and none required patient withdrawal from treatment or dose interruption.

The efficacy of RoActemra SC in children one to 17 years of age is based on PK exposure and extrapolation of established efficacy of RoActemra IV in sJIA patients and RoActemra SC in patients with RA.

The SC formulation of RoActemra, under the brand name Actemra, was recently approved by the US Food and Drug Administration for sJIA.

About Actemra /RoActemra (tocilizumab)

Actemra/RoActemra is the first 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 adult RA patients who are intolerant to, or have failed to respond to, other disease-modifying anti-rheumatic drugs (DMARDs). In Europe, RoActemra IV and SC are also approved for use in adult patients with severe, active and progressive RA who previously have not been treated with MTX. In children one year of age and older, Actemra/RoActemra is approved globally for IV and SC in polyarticular juvenile idiopathic arthritis (pJIA). It is also approved globally in the IV formulation, and in the US in the SC formulation for systemic juvenile idiopathic arthritis (sJIA). Actemra/RoActemra SC injection is also the first approved therapy for the treatment of giant cell arteritis (GCA) in more than 40 countries, including the US and Europe. Actemra was granted Breakthrough Therapy Designation for GCA by the US Food and Drug Administration (FDA) in October 2016. In the US and Europe, Actemra/RoActemra IV injection is approved for the treatment of chimeric antigen receptor (CAR) T-cell-induced severe or life-threatening cytokine release syndrome (CRS) in people two years of age and older. Actemra/RoActemra is the first approved treatment for CRS in this setting. In Japan, Actemra is also approved for the treatment of Castleman's Disease and Takayasu Arteritis. 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 more than 110 countries worldwide.

About Roche in rheumatology and beyond

For more than 50 years, Roche has followed the science to pioneer medicines for immune-mediated rheumatic diseases. First-in-class anti-IL-6 receptor therapy Actemra/RoActemra (tocilizumab) has treated more than one million people with debilitating conditions, such as rheumatoid arthritis (RA), polyarticular and systemic juvenile idiopathic arthritis, giant cell arteritis and chimeric antigen receptor T-cell-induced cytokine release syndrome. Rituxan/MabThera (rituximab), which targets CD20, has significant clinical and real-world experience treating rheumatic conditions including RA, granulomatosis with polyangiitis and microscopic polyangiitis. Roche aims to provide solutions for people that need new treatments most, particularly those with severe or life-threatening conditions and limited treatment options. Our pipeline consists of treatments designed to target immune pathways including a Bruton's tyrosine kinase inhibitor, which is being studied in RA, lupus erythematosus and chronic spontaneous urticaria, and a glycoengineered type II anti-CD20 antibody in lupus nephritis.

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 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.

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.
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. 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 tenth consecutive year, Roche has been recognised as the most sustainable company 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 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 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.