Wednesday, Jun 10, 2015

FDA Grants Breakthrough Therapy Designation for Actemra (tocilizumab) in Systemic Sclerosis

  • Systemic sclerosis affects approximately 75,000 to 100,000 people in the U.S. and has highest mortality rate of any autoimmune rheumatic disease(1,2)
  • There is no FDA-approved therapy for the rare disease(1)
  • Genentech initiates Phase III clinical trial

South San Francisco, CA -- June 10, 2015 --

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to Actemra® (tocilizumab) for systemic sclerosis, also known as scleroderma.1 This designation is intended to expedite the development and review of medicines with early signals of potential clinical benefit in serious diseases and to help ensure patients have access to them as soon as possible.  Genentech has also initiated a Phase III study in systemic sclerosis (NCT02453256), a disease for which there are inadequate treatment options.3,4

"The development of Actemra in systemic sclerosis represents our commitment to severe rheumatic diseases," said Sandra Horning, M.D., chief medical officer and head of Global Product Development.  "This Breakthrough Therapy Designation underscores the unmet need in systemic sclerosis and the potential of Actemra to help patients with this debilitating autoimmune disorder."

Actemra in systemic sclerosis

Systemic sclerosis is a rare, chronic disorder characterized by blood vessel abnormalities, as well as degenerative changes and scarring in the skin, joints, and internal organs.4 The disease affects approximately 75,000 to 100,000 people in the United States. More than 75 percent of systemic sclerosis patients are women, primarily aged 30 to 50.1 Systemic sclerosis has the highest mortality of any autoimmune rheumatic disease.2 

There is currently no FDA-approved treatment for systemic sclerosis. There are treatments for some aspects of this disease, but none are used to stop or reverse the key symptom of skin thickening and hardening.1 

The Breakthrough Therapy Designation for Actemra was granted based on data from the Phase II faSScinate study. Forty-eight week data will be presented as an oral presentation in Rome at EULAR 2015 (abstract number: OP0054; presentation date: Thursday, June 11, 2015).5 While the primary endpoint of improvement in skin thickening at 24 weeks – as assessed by the Rodnan skin score – was not met, a meaningful trend was observed. In this second part of the study, there was continued improvement in skin thickening between weeks 24 and 48.5  The overall adverse event profile between both groups was comparable.5 The extent and severity of skin thickness correlates to disease worsening, increased disability and decreased survival.6,7  Based on these Phase II results and the unmet need in patients with systemic sclerosis, Genentech initiated a multicenter, randomized, double-blind, placebo-controlled Phase III trial (NCT02453256). 

About Actemra

Actemra is the first humanized interleukin-6 (IL-6) receptor antagonist approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have used one or more disease-modifying antirheumatic drugs (DMARDs), such as methotrexate (MTX), that did not provide enough relief. The extensive Actemra RA IV clinical development program included five Phase III clinical studies and enrolled more than 4,000 people with RA in 41 countries, including the United States. The Actemra RA subcutaneous clinical development program included two Phase III clinical studies and enrolled more than 1,800 people with RA in 33 countries, including the United States. In addition, Actemra is also used as an IV formulation for patients with active polyarticular juvenile idiopathic arthritis (PJIA) or systemic juvenile idiopathic arthritis (SJIA) two years of age and older. 

Actemra is intended for use under the guidance of a healthcare practitioner.

Important Safety Information

Actemra can cause serious side effects. Actemra changes the way a patient’s immune system works. This can make a patient more likely to get infections or make any current infection worse. Some people taking Actemra have died from these infections. 

Actemra can cause other serious side effects. These include:

  • stomach tears
  • changes in blood test results, including low neutrophil (white blood cells) and platelet (platelets help the blood to clot) counts, and increases in certain liver function test levels and blood cholesterol levels
  • an increased risk of certain cancers by changing the way a patient’s immune system works
  • hepatitis B infection
  • serious allergic reactions, including death. These may happen with Actemra infusions or injections, even if they did not occur with an earlier infusion or injection.
  • nervous system problems

Patients should tell their doctor if they are allergic to Actemra or if they have had a bad reaction to Actemra previously.

Common side effects:

Patients should tell their doctor if they have these or any other side effect that bothers them or does not go away:

  • Upper respiratory tract infections (like common cold and sinus infections)
  • Headache
  • Increased blood pressure (also called hypertension)
  • Injection site reactions

Actemra & pregnancy:

Patients should tell their doctor if they are planning to become pregnant, are pregnant, plan to breast-feed, or are breast-feeding. The patient and their doctor should decide if the patient will take Actemra or breast-feed. Patients should not do both. Genentech has a registry for pregnant women who take Actemra. The purpose of this registry is to check the health of the pregnant mother and her baby. If a patient is pregnant or becomes pregnant while taking Actemra, she should talk to her healthcare provider about how she can join this pregnancy registry or she may contact the registry at 1-877-311-8972 to enroll. 

Patients should tell their doctor right away if they are experiencing any side effects. Report side effects to the FDA at 1-800-FDA-1088 or Call Genentech at 1-888-835-2555.

Please visit for the full Prescribing Information, including Boxed Warning and Medication Guide, for additional Important Safety Information or call 1-800-ACTEMRA (228-3672).

Actemra is part of a co-development agreement with Chugai Pharmaceutical Co. and has been approved in Japan since June 2005. Actemra is approved in the European Union, where it is known as RoActemra, and several other countries, including China, India, Brazil, Switzerland and Australia.

About Genentech

Founded more than 35 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit


1 The American College of Rheumatology. Scleroderma (also known as systemic sclerosis). Available at: [Last accessed: June 3, 2015]

2 Khanna D, Denton CP. Evidence-based management of rapidly progressing systemic sclerosis. Best Pract Res Clin Rheumatol 2010; 24: 387–400.

3 Krause L, et al. Nutritional status as marker for disease activity and severity predicting mortality in patients with systemic sclerosis. Ann Rheum Dis. 2010; 69: 1951–1957.

4 Hajj-ali R. Merck Manuals. Systemic Sclerosis. Available at: [Last accessed: May 13, 2015]

5 Khanna D, et al. Safety and efficacy of subcutaneous tocilizumab in adults with systemic sclerosis: week 48 data from the faSScinate trial. Oral presentation at EULAR, 2015. Abstract number: OP0054. Presentation date: Thursday, June 11, 2015. Presentation time: 10:35pm.

6 Clements PJ, et al. Skin thickness score as a predictor and correlate of outcome in systemic sclerosis. Arth and Rheum. 2000; 43(11): 2445-2454.

7 Steen VD, Medsger Jr TA. Improvement in skin thickening in systemic sclerosis associated with improved survival. Arth and Rheum. 2001; 44(12): 2828-2835.