Actemra (tocilizumab) is a medication by Genentech (a Roche subsidiary). The U.S. Food and Drug Administration (FDA) approved it to treat systemic sclerosis-associated interstitial lung disease (SSc-ILD).
How does Actemra work?
Systemic scleroderma is a rare autoimmune disease characterized by the thickening, hardening, and scarring of the skin. It is caused by the accumulation of excess collagen in the skin and internal organs, such as the heart, lungs, kidneys, and intestinal tract, leading to organ damage. An estimated 80% of patients have ILD due to the buildup of scar tissue and inflammation in the lungs, seriously affecting their ability to breathe
Actemra works by directly blocking the action of interleukin-6 (IL-6). Interleukins are proteins that white blood cells produce to regulate the immune response.
IL-6 plays a major role in the disease process of systemic scleroderma. Found at high levels in blood and skin lesions of patients with this condition, it appears to be associated with greater disease activity and higher mortality.
By preventing the activation of the immune system via the inhibition of IL-6, Actemra aims to lessen the excessive production of scar tissue in systemic scleroderma.
Actemra in clinical trials for systemic scleroderma
A Phase 2/3 clinical trial (NCT01532869), called faSScinate, investigated the efficacy and safety of Actemra in 87 patients with systemic scleroderma. During its first part, patients randomly received weekly injections of Actemra at 162 mg or a placebo for 48 weeks. During the trial’s second, open-label period, all received Actemra at 162 mg as under-the-skin injections for an additional 48 weeks.
Results from the first 48 weeks showed mild improvements in skin symptoms (the primary trial outcome) in patients receiving treatment compared to those on the placebo, although these changes were not significant. There was also evidence of better lung function among SSc-ILD patients receiving treatment with Actemra compared with those in the placebo group. However, there was no difference between the two groups in terms of disability, itching, fatigue, or overall disease severity.
Data from weeks 48 to 96 also showed that those taking Actemra were more likely to develop serious infections. However, there were no new side effects. Results from this open-label part of the trial also showed improved skin scores and stabilized lung function in people moving from placebo to treatment. Among patients continuing on Actemra, maintenance of benefits was evident.
A randomized, double-blind, and global focuSSced Phase 3 trial (NCT02453256) in 212 adult patients with systemic scleroderma began in 2015. Like the earlier trial, this study consisted of an initial 48-week period, where patients received either weekly injections of Actemra (162 mg) under the skin or a placebo. This was followed by an open-label period — weeks 48 to 96 — where all received Actemra.
The FDA approval is based on the results of the Roche-sponsored focuSSced study. It, too, did not meet its primary goal of a change in the modified Rodnan Skin Score (mRSS), a standard measure for skin thickness, comparing week 48 to the study’s start. mRSS lowered by a mean of 5.88 points in people who received Actemra and by 3.77 in those who received the placebo. This difference was not statistically significant.
However, a benefit was apparent in the 136 patients (68 in each group, treatment, and placebo) with confirmed SSc-ILD. Analyses found that these Actemra-treated patients, relative to those on placebo, had a smaller decline in mean forced vital capacity (FVC). FVC is a test of lung health that assesses how much air is exhaled after a deep breath. The mean FVC decline with Actemra was 14 mL and 255 mL with placebo after 48 weeks.
Actemra-treated patients also had a smaller decline in mean percent predicted FVC (ppFVC). Mean ppFVC compares FVC to that of a healthy person of the same age, sex, race, and height. Mean ppFVC in the Actemra group rose by 0.07%, compared to a 6.4% decline in the placebo group.
Because Actemra affects the immune system, it can cause side effects such as infections, including serious infections.
Patients who experience fever, chills, aches, tiredness, cough or skin sores, diarrhea or burning sensation with urination, or weight loss while using Actemra should notify their physician.
People with liver disease or those who have had tuberculosis or hepatitis should inform their doctor before starting treatment.
Actemra is also approved for people with rheumatoid arthritis, specific types of juvenile arthritis, and other indications. In Europe, it is available under the brand name RoActemra for rheumatoid arthritis and juvenile arthritis.
Last update: March 10, 2021
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