Investor Update

Basel, 26 November 2018

FDA approves the ACTPen for Roche’s Actemra, a single-dose, prefilled autoinjector for the treatment of rheumatoid arthritis, giant cell arteritis and two forms of juvenile arthritis

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the US Food and Drug Administration (FDA) has approved ACTPen™ 162 mg/0.9 mL, a single-dose prefilled autoinjector for Actemra®(tocilizumab) as an additional formulation for adult patients with moderate to severe active rheumatoid arthritis (RA) who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs (DMARDs), and for adult patients with giant cell arteritis (GCA). Further, the ACTPen can be administered by caregivers to patients two years of age and older with active polyarticular juvenile idiopathic arthritis (pJIA) or active systemic juvenile idiopathic arthritis (sJIA). The ability of pediatric patients to self-inject with the ACTPen has not been tested. ACTPen is expected to be available in January 2019.

“When it comes to the administration of medicines, we believe patients should have choices, when possible,” said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. “With ACTPen for Actemra, we are pleased to offer an additional option to patients who may prefer using the new autoinjector over other formulations.”

Actemra intravenous infusion formulation (IV) and Actemra pre-filled syringe (PFS) formulations for subcutaneous injection (SC) have been approved globally. In 2017, Actemra SC became the first therapy approved in the US and Europe for the treatment of adult patients with GCA, a chronic and severe form of vasculitis characterised by inflammation of certain large blood vessels. In addition, Actemra IV and SC have been approved globally for patients two years of age and older. ACTPen received EU approval for patients with RA and GCA in May 2018. Since 2010, more than one million patients have been treated with Actemra worldwide.

The approval of the ACTPen is based on clinical data from two studies that were presented at the 2018 American Society for Clinical Pharmacology & Therapeutics Annual Meeting.[1] The first was an open-label, randomised, two-period, crossover phase I study, investigating the relative bioavailability of a single injection of Actemra 162 mg SC via the PFS with needle safety device to a single injection of Actemra 162 mg SC via the ACTPen in 188 healthy volunteers. The second was an open-label, non-randomised, observational phase IV human factors study in 54 adult patients with RA investigating whether the ACTPen could be used safely and effectively by patients, caregivers or health care professionals to administer the Actemra injection. The studies found that the single-dose SC administration of 162 mg Actemra with the ACTPen was bioequivalent to administration with the currently marketed PFS, and the intended users of the ACTPen were successful in performing the tasks required to administer doses of Actemra. The adverse events of Actemra in both studies were consistent with the medicine's established safety profile.

About Rheumatoid Arthritis

Rheumatoid arthritis (RA) is a chronic, progressive inflammatory autoimmune disease of the joints and surrounding tissues, associated with swelling in and pain around the joints.[2] The condition impacts approximately 35-70 million people worldwide.[3,4] If left untreated, RA can lead to irreversible joint destruction and systemic complications.[2]

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, usually affects those above the age of 50, and is two-to-three-times more likely to affect women than men.[5] GCA is often difficult to diagnose because of the wide and variable spectrum of signs and symptoms. GCA can cause severe headaches, scalp tenderness, jaw pain and visual symptoms and if left untreated, can lead to blindness, aortic aneurysm or stroke.[5] 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, in some cases steroids are unable to maintain long-term disease control (flare-free remission).[6,7,8] Due to the variability of symptoms, complexity of the disease and disease complications, people with GCA are often seen by several physicians including rheumatologists, ophthalmologists and neurologists.

About Polyarticular Juvenile Idiopathic Arthritis and Systemic Juvenile Idiopathic Arthritis

Polyarticular juvenile idiopathic arthritis (pJIA) and systemic juvenile idiopathic arthritis (sJIA) are forms of juvenile idiopathic arthritis (JIA), a chronic arthritic disease affecting children.[9] JIA affects nearly 100 per 100,000 children globally of which pJIA accounts for around 25% and sJIA accounts for around 10-20%.[9,10] pJIA is characterised by inflammation in five or more joints within the first six months of the disease and most commonly affects the small joints in the body such as the hands and feet.[9] sJIA is characterized 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.[9] Common symptoms include pain, tenderness, swelling and warmth in the joints.[9]

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. Actemra/RoActemra IV and SC are approved globally for polyarticular juvenile idiopathic arthritis (pJIA) and in the US and Europe for systemic juvenile idiopathic arthritis (sJIA) in children two years of age and older. 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 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. A prefilled autoinjector ACTPen has been approved in the US and Europe for patients with RA, GCA, sJIA and pJIA. 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.

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

References

  1. Fettner S et al. Evaluation of a Tocilizumab Autoinjector: Results of a Healthy Volunteer Bioequivalence and Rheumatoid Patient Human Factors Study, Poster Presentation, 2018 ASCPT Annual Meeting; March 21–24, 2018; Orlando, Florida.
  2. Patient UK. Rheumatoid arthritis. [Internet; cited 2018 Nov]. Available at: http://www.patient.co.uk/ health/rheumatoid-arthritis-leaflet.
  3. Symmons D, et al. The global burden of rheumatoid arthritis in the year 2000. [Internet; cited 2018 Jan]. Available at: http://www.who.int/healthinfo/statistics/bod_rheumatoidarthritis.pdf.
  4. Gabriel SE, et al. Epidemiological studies in incidence, prevalence, mortality, and comorbidity of the rheumatic diseases. Arthritis Res Ther. 2009; 11:229.
  5. Bhat S, et al. Giant cell arteritis. Midlife and Beyond, GM. Rheumatology. 2010; 071-079.
  6. Balsalobre A, et al. Temporal Arteritis: Treatment Controversies. Neurologia. 2010; 25(7): 453-458.
  7. Ponte C, et al. Giant cell arteritis: Current treatment and management. World J Clin Cases. 2015; 3(6): 484-494.
  8. Chatterjee S, et al. Clinical Diagnosis and Management of Large Vessel Vasculitis: Giant Cell Arteritis. Curr Cardiol Rep. 2014; 16:498.
  9. Arthritis Foundation. Juvenile Idiopathic Arthritis. [Internet; cited 2018 Nov]. Available at: https://www.arthritis.org/about-arthritis/types/juvenile-idiopathic-arthritis-jia/what-is-juvenile-idiopathic-arthritis.php.
  10. Woo P. Systemic juvenile rheumatoid arthritis: diagnosis, management, and outcome. Nat Clin Pract Rheumatol. 2006; 2(1):28-34.