FDA Approves ACTEMRA® (tocilizumab) for the Treatment of Systemic Juvenile Idiop

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April 15, 2011 6:04 PM EDT

– Medicine offers a new option for children living with a rare and severe form

of arthritis –

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche

Group (SIX: RO, ROG; OTCQX: RHHBY), 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 & #8805;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)