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Tuesday, Apr 30, 2013

FDA Approves ACTEMRA® for Children Living with a Rare Form of Arthritis

Medicine Offers a New Option for the Treatment of Polyarticular Juvenile Idiopathic Arthritis (PJIA)

South San Francisco, Calif. -- April 30, 2013 -- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved ACTEMRA® (tocilizumab) for the treatment of polyarticular juvenile idiopathic arthritis (PJIA). The medicine can be used in children two years of age and older with active disease. ACTEMRA can be given alone or in combination with methotrexate (MTX) in people with PJIA.


PJIA is a form of juvenile idiopathic arthritis (JIA), also known as juvenile rheumatoid arthritis, a chronic disease of childhood.1 JIA affects approximately 100 in every 100,000 children2 of which PJIA accounts for around 30 percent.3 PJIA is characterized 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.3


"Polyarticular juvenile idiopathic arthritis is a rare, debilitating condition in children that worsens over time," said Hal Barron, M.D., chief medical officer and head, Global Product Development. "We are pleased to offer ACTEMRA to doctors and parents of children aged two or older as an important medicine to help improve the signs and symptoms of this often painful disease."


This FDA approval marks the second ACTEMRA indication in children and is the first FDA approval for the treatment of PJIA in approximately five years.


About the CHERISH Study
The expanded indication for ACTEMRA was based on positive data from the Phase III CHERISH study in children with PJIA, which had an open label phase, followed by a randomized double-blind placebo-controlled withdrawal phase. The study demonstrated that patients treated with ACTEMRA experienced clinically meaningful improvement in signs and symptoms of PJIA. A total of 91 percent of patients taking ACTEMRA plus MTX and 83 percent of patients taking ACTEMRA alone achieved an ACR 30 response at week 16 compared to baseline. In the randomized double-blind placebo-controlled withdrawal phase of the trial, ACTEMRA-treated patients experienced significantly fewer disease flares compared to placebo-treated patients (26 percent [21/82] vs. 48 percent [39/81]).


The safety data collected to date for ACTEMRA in PJIA patients is consistent with that observed in previous studies in ACTEMRA-treated patients.4 In the CHERISH study, infections were the most common adverse events (AEs) and serious adverse events (SAEs) over 40 weeks. Laboratory abnormalities known to occur with ACTEMRA were also observed in this study, including decreases in white blood cell counts and platelet counts, and elevation in ALT and AST liver enzyme levels.


About JIA ACR 30, 50, 70
The JIA American College of Rheumatology (ACR) response was derived from the following six variables:

  • Parents/Patients Global Assessment of Overall Well-Being
  • Physicians Global Assessment of Disease Activity
  • Number of joints with active arthritis
  • Number of joints with limitation of movement
  • Acute phase reactant (erythrocyte sedimentation rate (ESR))
  • Functional ability determined by Childhood Health Assessment Questionnaire (C-HAQ) disability index (~xr30i)

About ACTEMRA® (tocilizumab)
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 had an inadequate response to one or more disease-modifying antirheumatic drugs (DMARDs). The extensive ACTEMRA RA 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 also approved for the treatment of active systemic juvenile idiopathic arthritis (SJIA) and polyarticular juvenile idiopathic arthritis (PJIA) in patients two years of age and older.


Important Safety Information
Some people developed 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 (including low white blood cell count, low platelet count, increase in certain liver function tests, and increase in blood cholesterol levels), hepatitis B infection becoming an active infection again, and nervous system problems.


ACTEMRA affects the immune system and may increase a patient's risk of certain cancers.


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 in patients with RA include upper respiratory tract infections (common cold, sinus infections), headache, and increased blood pressure (hypertension).


Common side effects with ACTEMRA in patients with PJIA or SJIA include upper respiratory tract infections (common cold, sinus infections), headache, and diarrhea.


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.


Report side effects to the FDA at 1-800-FDA-1088 or http://www.FDA.gov/medwatch. Patients and caregivers may also report side effects to Genentech at 1-888-835-2555.


Please visit http://www.actemra.com 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 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, Calif. For additional information about the company, please visit http://www.gene.com.


References
1 Beukelman, T et al. 2011 American College of Rheumatology Recommendations for the Treatment of Juvenile Idiopathic Arthritis: Initiation and Safety Monitoring of Therapeutic Agents for the Treatment of Arthritis and Systemic Features. Arthritis Care & Research. 2011.http://www.ncbi.nlm.nih.gov/pmc/articles/PMC32222233/. Accessed April 2, 2013.
2 Woo, P. Systemic Juvenile Rheumatoid Arthritis: Diagnosis, Management, and Outcome. Nature Clinical Practice: Rheumatology. 2006. 2:1.
3 Macaubas, Claudia et al. Oligoarticular and Polyarticular JIA: Epidemiology and Pathogenesis. Nature Reviews. 2009 vl 5.
4 De Benedetti F, et al. Ann Rheum Dis 2011; 70 (Suppl. 3):67.

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