FDA Approves Actemra for Systemic Juvenile Idiopathic Arthritis

FDA Approves Actemra (tocilizumab) for the Treatment of Systemic Juvenile Idiopathic Arthritis (SJIA)

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Apr 15, 2011 - Genentech, a member of the Roche Group today announced that the U.S. Food and Drug Administration (FDA) approved Actemra (tocilizumab) for the treatment of active Systemic Juvenile Idiopathic Arthritis (SJIA) in patients two years of age and older. Actemra can be given alone or in combination with methotrexate in patients with SJIA.

Actemra is the first medicine approved by the FDA for the treatment of SJIA, a rare and severe form of arthritis affecting children. SJIA has the worst long-term prognosis of all types of childhood arthritis.1

"Today's FDA approval marks an important advance in the treatment of SJIA, a debilitating condition affecting children," said Hal Barron, M.D., chief medical officer and head Global Product Development. "As the first and only approved treatment for SJIA, Actemra offers a new option for this extremely difficult to treat disease. This approval also demonstrates our commitment to science and patients with high unmet medical need, including orphan diseases."

"The goal of treatment for children with SJIA is to reduce the signs and symptoms of the disease, including swelling, pain and other complications," said Hermine Brunner, M.D., MSc, associate professor of pediatric rheumatology, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center, scientific director of the pediatric rheumatology collaborative study group, and a study investigator. "We're excited about the results of this study which show that Actemra significantly improved disease signs and symptoms as measured by a JIA ACR response, plus absence of fever, a critical validated efficacy measure of SJIA treatment."

SJIA is the rarest form of Juvenile Idiopathic Arthritis (JIA), also known as Juvenile Rheumatoid Arthritis (JRA).2 The disease affects about 10 to 20 percent of children with JIA,3 with the peak age of onset between 18 months and two years,3,4 although the disease can persist into adulthood. SJIA has a two to four percent overall estimated mortality rate, and accounts for almost two-thirds of all deaths among children with arthritis.1 The severity of SJIA varies from person to person and can include symptoms ranging from joint inflammation accompanied by intermittent fever, skin rash, anemia, enlargement of the liver or spleen and inflammation of the lining of the heart and/or lungs.5 In the most severe cases of SJIA, up to two-thirds of children experience chronic arthritis, and approximately half of children will develop significant joint disabilities.6,7

About the TENDER Study

This approval was based on positive data from a Phase III study known as TENDER. The results showed that 85 percent (64/75) of children with SJIA receiving Actemra experienced a 30 percent improvement (JIA ACR30) in the signs and symptoms of SJIA and an absence of fever after 12 weeks of therapy, compared with 24 percent (9/37) of children receiving placebo (p<0.0001).8

Additional results from the TENDER study, a randomized, double-blind, Phase III study in 112 patients showed significantly more children who received Actemra had improvements in SJIA signs and symptoms. In the study, 71 percent (53/75) of children treated with Actemra achieved a JIA ACR70 response at week 12 compared with eight percent (3/37) of those receiving placebo (p<0.0001).

No new or unexpected safety signals were identified with Actemra.8 The most common adverse events (at least five percent) seen in Actemra treated patients in the 12 week controlled portion of the study were upper respiratory tract infection, headache, nasopharyngitis and diarrhea. The most commonly reported serious infections included pneumonia, gastroenteritis, varicella (chickenpox) and otitis media (ear infection). Sixteen percent of patients in the Actemra treatment group and five percent of patients in the placebo group experienced an infusion reaction. Anaphylaxis was reported in one of the 112 patients treated with Actemra during the controlled and open-label extension study.

This Phase III international study included 43 sites in 17 countries. The study evaluated the efficacy and safety profile of Actemra versus placebo over 12 weeks in 112 children with SJIA. This study is the first part of a five-year ongoing study.

Patients two to 17 years of age with active SJIA for at least six months (average disease duration in the study was five years) who could not tolerate, or did not respond well to their current therapy (NSAIDs and/or systemic corticosteroids) were randomized to receive Actemra (8 mg/kg if weight ‰¥30 kg or 12 mg/kg if weight <30 kg) or placebo every two weeks as a 60-minute single intravenous drip infusion for a total of 12 weeks. Patients continued to receive NSAIDs, corticosteroids and methotrexate if receiving these medicines at the start of the study. The primary endpoint was the number of patients treated with Actemra with a JIA ACR30 response and absence of fever at week 12, compared with those receiving placebo.

About Actemra (tocilizumab)

Actemra is the first humanized IL-6 receptor-inhibiting monoclonal antibody approved for the treatment of adult patients with moderately to severely active RA who have had an inadequate response to one or more tumor necrosis factor (TNF) antagonist therapies. The extensive Actemra 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. In addition, Actemra is now approved for the treatment of active SJIA in patients two years of age and older.

Some people have serious infections while taking Actemra, including tuberculosis (TB), and infections caused by bacteria, fungi, or viruses that can spread throughout the body. Some people have died from these infections. Other serious side effects of Actemra include tears (perforation) of the stomach and intestines, changes in blood test results, hepatitis B infection becoming an active infection again, and nervous system problems. Serious allergic reactions, including death, can happen with Actemra. These reactions may happen with any infusion of Actemra even if they did not occur with an earlier infusion. Patients must tell their doctor if they have had a previous reaction to Actemra. Patients should not take Actemra if they are allergic to it or any of its ingredients.

Common side effects with Actemra include upper respiratory tract infections (common cold, sinus infections), headache, and increased blood pressure (hypertension). Patients must tell their healthcare providers if they plan to become pregnant or are pregnant. It is not known if Actemra will harm an unborn baby. Genentech has a registry for pregnant women who take Actemra. Patients who are pregnant or become pregnant while taking Actemra must contact the registry at 1-877-311-8972 and talk to their healthcare provider.

Patients must call their healthcare provider for medical advice about any side effects. Patients or caregivers may report side effects to the FDA at 1-800-FDA-1088. Patients or caregivers may also report side effects to Genentech at 1-888-835-2555.

For additional important safety information, including Boxed WARNINGS and Medication Guide, please visit http://www.actemra.com 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 India, Brazil, Switzerland and Australia.

About Genentech

Founded more than 30 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 http://www.gene.com.

1 Cassidy JT, et al. Juvenile rheumatoid arthritis. Cassidy JT, Petty RE, eds. Textbook of pediatric rheumatology 2001:218-322.

2 Fishman D, Faulds G, Jeffery R, et al. The effect of novel polymorphisms in the interleukin-6 (IL-6) gene on IL-6 transcription and plasma IL-6 levels, and an association with systemic-onset juvenile chronic arthritis. J Clin Invest. 1998;102:1369-1376.

3 Rabinovich CE. Juvenile Rheumatoid Arthritis. Medscape eMedicine Rheumatology. http://emedicine.medscape.com/article/1007276-overview. Updated June 1, 2010. Accessed March 1, 2010.

4 Symmons DP et al. Pediatric rheumatology in the United Kingdom: data from the British Paediatric Rheumatology Group National Diagnostic Register. J Rheumatology. 1996: 23: 1975-1980.

5 Woo P. Systemic juvenile rheumatoid arthritis: diagnosis, management, and outcome. Nature Clinical Practice: Rheumatology. 2006. 2:1

6 Lomater C, Gerloni V, Gattinara M, Mazzotti J, Cimaz R, Fantini F. Systemic onset juvenile idiopathic arthritis: a retrospective study of 80 consecutive patients followed for 10 years. J Rheumatol. 2000;27(2):491-6.

7 Prieur AM, Brémard-Oury C, Griscelli C, Mozziconacci P. Prognosis of the systemic forms of juvenile chronic arthritis. Apropos of 100 cases. Arch Fr Pediatr. 1984;41(2):91-7.

8 Tocilizumab in Patients With Systemic Juvenile Idiopathic Arthritis: Efficacy Data From the Placebo-Controlled 12-Week Part of the Phase 3 TENDER Trial. Abstract presented at ACR 2010.

Contact: Genentech
Joe St. Martin, 650-467-6800 (Media)
Karl Mahler, 011 41 61 687 8503 (Investors)
Thomas Kudsk Larsen, 973-235-3655 (Investors)

Posted: April 2011

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