FDA Approves Subcutaneous Formulation of Actemra for Use in Active Polyarticular Juvenile Idiopathic Arthritis (PJIA), a Rare Form of Juvenile Arthritis

South San Francisco, CA -- May 14, 2018 -- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved the subcutaneous (SC) formulation of Actemra® (tocilizumab) for the treatment of active polyarticular juvenile idiopathic arthritis (PJIA) in patients two years...

South San Francisco, CA -- May 14, 2018 --

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved the subcutaneous (SC) formulation of Actemra® (tocilizumab) for the treatment of active polyarticular juvenile idiopathic arthritis (PJIA) in patients two years of age and older. Actemra can be given alone or in combination with methotrexate (MTX) in patients with PJIA. In 2013, FDA approved the intravenous (IV) formulation of Actemra for patients two years of age and older with active PJIA.

PJIA is a form of juvenile idiopathic arthritis (JIA), a chronic disease of childhood.1 JIA affects approximately 100 in every 100,000 children,2 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, often painful disease in children," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "With this approval, we are pleased Actemra offers an alternative delivery option to physicians and parents of children aged two or older to treat this debilitating disease."

The approval is based on data from the JIGSAW-117 study, a 52-week, open-label, multicenter, phase 1b pharmacokinetic (PK)/pharmacodynamic (PD) bridging study designed to determine the appropriate dosing regimen of Actemra SC across a range of body weights (BWs) in children with PJIA.4 The study enrolled 52 patients aged one to 17 years with PJIA and previous inadequate response or intolerance to methotrexate who were either Actemra naive or were receiving Actemra IV with adequate disease control. Actemra SC was administered open label according to a body weight (BW)–based dosing regimen: PJIA patients weighing

In general, the safety observed for Actemra administered subcutaneously was consistent with the known safety profile of Actemra IV, with the exception of injection site reactions (ISRs) and neutropenia (low white blood cell count). A higher frequency of ISRs was observed in Actemra SC treated PJIA patients compared to patients treated with Actemra SC for other approved indications. During the duration of the one-year study, a frequency of 28.8% (15/52) ISRs was observed in Actemra SC treated PJIA patients. All ISRs reported were mild in severity, and none of the ISRs required patient withdrawal from treatment or dose interruption. During routine laboratory monitoring in Actemra SC treated PJIA patients, a decrease in neutrophil counts below 1 × 109 per L occurred in 15.4% of patients, and was more frequently observed in the patients less than 30 kg (25.9%) compared to patients at or above 30 kg (4.0%). Neutrophils are a type of white blood cell.

The efficacy of Actemra SC in children two to 17 years of ages is based on PK exposure and extrapolation of established efficacy of Actemra IV in PJIA patients and Actemra SC in patients with RA.

About Actemra

Actemra was 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. 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. Actemra subcutaneous injection is also approved for the treatment of adult patients with giant cell arteritis (GCA) and for patients two years of age and older with active polyarticular juvenile idiopathic arthritis (PJIA). In addition, Actemra is also used as the IV formulation for patients two years of age and older with active PJIA, systemic juvenile idiopathic arthritis (SJIA) or CAR T cell-induced cytokine release syndrome (CRS). Actemra is not approved for subcutaneous use in people with SJIA or CRS. It is not known if Actemra is safe and effective in children with PJIA, SJIA or CRS under two years of age or in children with conditions other than PJIA, SJIA or CRS.

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:

  • Tears of the stomach or intestines
  • 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. If a patient has had hives, a rash, or experienced flushing after injecting, the patient should tell their doctor or nurse before their next injection
  • Nervous system problems

Patients should not receive Actemra if they are allergic to Actemra or if they have had a bad reaction to Actemra previously.

Most common side effects in patients treated with Actemra:

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 breastfeed, or are breastfeeding. The patient and their doctor should decide if the patient will take Actemra or breastfeed. Patients should not do both. If a patient is pregnant and taking Actemra, they should join the pregnancy registry. To learn more, patients should call 1-877-311-8972 or talk to their doctor to register.

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 http://www.FDA.gov/medwatch. 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 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and 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.

References
1Beukelman, 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.
2Woo, P. Systemic Juvenile Rheumatoid Arthritis: Diagnosis, Management, and Outcome. Nature Clinical Practice: Rheumatology. 2006. 2:1.
3Macaubas, Claudia et al. Oligoarticular and Polyarticular JIA: Epidemiology and Pathogenesis. Nature Reviews. 2009 vl 5.
4Brunner H et al. “Identification of Optimal Subcutaneous Doses of Tocilizumab in Children With Polyarticular-Course Juvenile Idiopathic Arthritis.” PRSYM 2017 (Pediatric Rheumatology Symposium); May 17–20, 2017; Houston, Texas.

Source: www.gene.com