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

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- 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 systemic juvenile idiopathic arthritis (SJIA) in patients two years of age and older. Actemra can be given alone or in combination with methotrexate (MTX) in patients with SJIA. In 2011, FDA approved the intravenous (IV) formulation of Actemra for patients two years of age and older with active SJIA.

“Systemic juvenile idiopathic arthritis is a rare, debilitating disease with limited treatment options,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are pleased to now offer physicians the flexibility to prescribe for children two years of age and older either Actemra IV, administered in a medical office, or Actemra subcutaneous, a prefilled syringe that can be injected at home.”

SJIA is the rarest form of juvenile idiopathic arthritis (JIA), a chronic arthritic disease affecting children.¹ JIA affects nearly 300,000 children in the U.S., of which SJIA accounts for around 10 percent. SJIA is characterized by inflammation in one or more joints, and a daily, spiking fever for at least two weeks, which may be accompanied by a skin rash.¹ Other symptoms may include anemia, enlargement of the liver or spleen, and inflammation of the lining of the heart and/or lungs.¹

The approval is based on data from the JIGSAW-118 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 SJIA.² The study enrolled 51 patients aged one to 17 years with SJIA and inadequate response to NSAIDs and corticosteroids 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 –based dosing regimen: SJIA patients weighing <30 kg received 162 mg of Actemra every two weeks or 10 days; and SJIA patients weighing ≥30 kg received 162 mg of Actemra every week for 52 weeks. Model-computed PK and PD parameters, and safety were assessed.

In general, the safety observed for Actemra SC was consistent with the known safety profile of Actemra IV, with the exception of injection site reactions (ISRs). A higher frequency of Actemra SC treated patients experienced ISRs, 41 percent (21/51), compared to patients treated with Actemra SC for other approved indications. All ISRs reported were non-serious, and none required patient withdrawal from treatment or dose interruption.

The efficacy of Actemra SC in children two to 17 years of age is based on PK exposure and extrapolation of established efficacy of Actemra IV in SJIA patients and Actemra SC in patients with rheumatoid arthritis (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) or active systemic juvenile idiopathic arthritis (SJIA). In addition, Actemra is also used as the IV formulation for patients two years of age and older with active PJIA, SJIA or CAR T cell-induced cytokine release syndrome (CRS). Actemra is not approved for subcutaneous use in people with 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

¹ Arthritis Foundation. https://www.arthritis.org/about-arthritis/types/juvenile-idiopathic-arthritis-jia/what-is-juvenile-idiopathic-arthritis.php (Last accessed: July 19, 2018)

² Brunner H et al. OP0103 Identification of optimal subcutaneous doses of tocilizumab in children with systemic juvenile idiopathic arthritis. Annals of the Rheumatic Diseases 2018;77:102.

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