Roche: Genentech’s Actemra Becomes the First Biologic Therapy Approved by the FDA for Slowing the Rate of Decline in Pulmonary Function in Adults With Systemic Sclerosis-Associated Interstitial Lung Disease, a Rare, Debilitating Condition Freitag, 05. März 2021 - 05:31
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Genentech’s Actemra Becomes the First Biologic Therapy Approved by the FDA for Slowing the Rate of Decline in Pulmonary Function in Adults With Systemic Sclerosis-Associated Interstitial Lung Disease, a Rare, Debilitating Condition
Systemic sclerosis (SSc) is a rare disease that impacts up to 75,000 people in the United States Approximately 80% of SSc patients may be affected by interstitial lung disease (ILD), a progressive disease that can significantly impact lung function and can be life-threatening In a global study, Actemra reduced the rate of progressive loss of lung function in people with SSc-ILD compared to placebo The U.S. Food and Drug Administration previously granted Priority Review designation to Actemra for the treatment of SSc-ILD SOUTH SAN FRANCISCO, CA -- March 4, 2021 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) approved Actemra® (tocilizumab) subcutaneous injection for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD), a debilitating condition with limited treatment options. Actemra is the first biologic therapy approved by the FDA for the treatment of the disease. Systemic sclerosis (SSc), also known as scleroderma, is an often devastating autoimmune disease that worsens over time and has no cure. It occurs when the immune system malfunctions causing tissues of the skin and lungs to thicken and harden. SSc impacts up to 75,000 people in the United States. Interstitial lung disease (ILD), which may occur in approximately 80% of SSc patients, causes inflammation and scarring of the lungs and can be life-threatening. “We are honored to offer the very first FDA-approved biologic treatment option to people living with systemic sclerosis-associated interstitial lung disease,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We worked closely with the FDA to evaluate Actemra’s impact on lung function in this setting. This milestone approval provides a much-needed new treatment option for people living with this rare, debilitating disease.” The FDA approval is based on data from the focuSSced trial, a Phase III randomized, double-blind, placebo-controlled clinical trial of 212 adults with systemic sclerosis. Supportive information was also used from the faSScinate trial, a Phase II/III, randomized, double-blind, placebo-controlled study in patients with SSc. The trials did not meet their primary endpoint of change from baseline to week 48 in the modified Rodnan Skin Score (mRSS), which is a standard outcome measure for skin fibrosis (the scarring or hardening of the skin) in SSc. However, in the overall population of the focuSSced study, patients treated with Actemra, as compared to placebo-treated patients, were observed to have less decline from baseline to week 48 in observed forced vital capacity (FVC), a common measure of lung function that assesses how much air can be exhaled, and percent predicted forced vital capacity (ppFVC), which compares the observed FVC to that expected for a healthy person of the same age, gender, race and height. FVC results were similar in the faSScinate study. Of the 212 patients who were randomized into the focuSSced study, 68 patients (65%) in the Actemra arm and 68 patients (64%) in the placebo arm had SSc-ILD at baseline, as confirmed by a visual read of high resolution computed tomograph (HRCT) by blinded thoracic radiologists. Post-hoc exploratory analyses were performed to evaluate the results within the subgroups of patients with and without SSc-ILD. The ppFVC and FVC results in the overall population were primarily driven by results in the SSc-ILD subgroup. In that subgroup, patients in the Actemra group had a smaller decline in mean ppFVC than patients on placebo (0.07% vs. -6.4%, mean difference 6.47%), and a smaller decline in FVC compared to placebo (mean change -14 mL vs. -255 mL, mean difference 241 mL). The mean change from baseline to week 48 in mRSS in patients receiving Actemra compared to placebo was -5.88 vs. -3.77, mean difference -2.11. The safety profile for Actemra through week 48 was comparable for SSc-ILD and SSc patients overall in the focuSSced study, and was consistent with the known safety profile of Actemra in both focuSSced and faSScinate studies. The most common adverse events in patients treated with Actemra were infections. Actemra was previously granted Priority Review for this condition by the FDA. This designation is given to medicines that have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease. This is the sixth FDA approved indication for Actemra since the medicine was launched in the United States in 2010. About the focuSSced Trial The primary efficacy endpoint was change from baseline at week 48 in mRSS. Change from baseline in FVC at week 48 was a key secondary endpoint. Of the 212 patients who were randomized, 68 patients (65%) in the Actemra arm and 68 patients (64%) in the placebo arm had SSc-ILD at baseline, as confirmed by a visual read of high resolution computed tomograph (HRCT) by blinded thoracic radiologists. The mean ppFVC at baseline for patients with SSc-ILD identified by HRCT was 79.6% (median 80.5%). Post-hoc analyses were performed to evaluate results within the subgroups of patients with and without SSc-ILD. The results of the key FVC secondary endpoints support the effectiveness of Actemra in reducing the rate of progressive loss of lung function in SSc-ILD patients. However, as the trial did not provide evidence of an effect on the primary endpoint of mRSS, the estimated magnitude of effect on the FVC endpoints should be interpreted with caution and comparisons to results of other products and studies may be misleading. About Actemra Actemra is intended for use under the guidance of a healthcare practitioner. Important Safety Information Actemra can cause other serious side effects. These include:
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:
Actemra & pregnancy: 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). About Genentech Access Solutions About Genentech ### |