FDA Approves the ACTPen for Genentech’s Actemra, a Single-Dose, Prefilled Autoinjector for the Treatment of Rheumatoid Arthritis, Giant Cell Arteritis and Two Forms of Juvenile Arthritis

SOUTH SAN FRANCISCO, Calif.

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 ACTPen™ 162 mg/0.9 mL, a single-dose prefilled autoinjector for Actemra^® (tocilizumab) as an additional formulation for adult patients with moderate to severe active rheumatoid arthritis (RA) who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs (DMARDs), and for adult patients with giant cell arteritis (GCA). Further, the ACTPen can be administered by caregivers to patients two years of age and older with active polyarticular juvenile idiopathic arthritis (PJIA) or active systemic juvenile idiopathic arthritis (SJIA). The ability of pediatric patients to self-inject with the ACTPen has not been tested. ACTPen is expected to be available in January 2019.

“When it comes to the administration of medicines, we believe patients should have choices, when possible,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “With ACTPen for Actemra, we are pleased to offer an additional option to patients who may prefer using the new autoinjector over other formulations.”

The FDA first approved Actemra intravenous infusion formulation (IV) for adults with RA in January 2010 and Actemra pre-filled syringe (PFS) formulations for subcutaneous injection (SC) for adults with RA in October 2013. In May 2017, Actemra SC became the first therapy approved by the FDA for the treatment of adult patients with GCA, a chronic and severe form of vasculitis characterized by inflammation of certain large blood vessels. Actemra IV was approved by the FDA for patients two years of age and older with active SJIA in April 2011 and active PJIA in April 2013, and Actemra SC was approved for these indications earlier this year. Since 2010, more than one million patients have been treated with Actemra worldwide.

The approval of the ACTPen is based on clinical data from two studies that were presented at the 2018 American Society for Clinical Pharmacology & Therapeutics Annual Meeting.¹ The first was an open-label, randomized, two-period, crossover Phase I study, investigating the relative bioavailability of a single injection of Actemra 162 mg SC via the PFS with needle safety device to a single injection of Actemra 162 mg SC via the ACTPen in 188 healthy volunteers. The second was an open-label, non-randomized, observational Phase IV human factors study in 54 adult RA patients investigating whether the ACTPen could be used safely and effectively by patients, caregivers or health care professionals to administer the Actemra injection. The studies found that the single-dose SC administration of 162 mg Actemra with the ACTPen was bioequivalent to administration with the currently marketed PFS, and the intended users of the ACTPen were successful in performing the tasks required to administer doses of Actemra. The adverse events of Actemra in both studies were consistent with the medicine’s established safety profile.

About Rheumatoid Arthritis

Rheumatoid arthritis (RA) is a chronic, progressive inflammatory autoimmune disease of the joints and surrounding tissues, associated with swelling in and pain around the joints.² The condition impacts approximately 1.5 million people in the U.S., with nearly three-quarters of the prevalence occurring in women.² If left untreated, RA can lead to irreversible joint destruction and systemic complications.²

About Giant Cell Arteritis

Giant cell arteritis (GCA), also known as temporal arteritis, affects an estimated 228,000 people over the age of 50 in the U.S., and the disease is two to three times more likely to affect women than men.^3,4 GCA can cause severe headaches, jaw pain and visual symptoms and, if left untreated, can lead to blindness, aortic aneurysm or stroke.⁴

About Polyarticular Juvenile Idiopathic Arthritis and Systemic Juvenile Idiopathic Arthritis

Polyarticular juvenile idiopathic arthritis (PJIA) and systemic juvenile idiopathic arthritis (SJIA) are forms of juvenile idiopathic arthritis (JIA), a chronic arthritic disease affecting children.⁵ JIA affects nearly 300,000 children in the U.S., of which PJIA accounts for around 25 percent and SJIA accounts for around 10 percent.⁵ 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.⁶ 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.⁵ Other symptoms may include anemia, enlargement of the liver or spleen, and inflammation of the lining of the heart and/or lungs.⁵

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 approved in 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. Fettner S et al. Evaluation of a Tocilizumab Autoinjector: Results of a Healthy Volunteer Bioequivalence and Rheumatoid Patient Human Factors Study, Poster Presentation, 2018 ASCPT Annual Meeting; March 21–24, 2018; Orlando, Florida.

2. Arthritis Foundation. What is Rheumatoid Arthritis? https://www.arthritis.org/about-arthritis/types/rheumatoid-arthritis/what-is-rheumatoid-arthritis.php (Last accessed: October 2018)

3. Lawrence RC, et al. Estimates of the prevalence of arthritis and other rheumatic conditions in the United States, Part II. Arthritis Rheum. 2008; 58: 26-35.

4. Bhat S, et al. Giant cell arteritis. Midlife and Beyond, GM. Rheumatology. February 2010; 071-079.

5. Arthritis Foundation. https://www.arthritis.org/about-arthritis/types/juvenile-idiopathic-arthritis-jia/what-is-juvenile-idiopathic-arthritis.php (Last accessed: November 14, 2018)

6. Macaubas, Claudia et al. Oligoarticular and Polyarticular JIA: Epidemiology and Pathogenesis. Nature Reviews. 2009 vl 5

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