-- Systemic sclerosis (SSc) is a rare disease that affects about 2.5 million 
      people worldwide 
 
   -- 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/RoActemra 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/RoActemra for the treatment of SSc-ILD 
 
   Basel, 05 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today 
announced that the U.S. Food and Drug Administration (FDA) approved 
Actemra(R) /RoActemra(R) (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/RoActemra 
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.(1-3) SSc affects about 2.5 million 
people worldwide.(4) 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.(5) 
 
   "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., Roche's Chief Medical 
Officer and Head of Global Product Development. "We worked closely with 
the FDA to evaluate Actemra/RoActemra'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 
randomised, 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, randomised, double-blind, 
placebo-controlled study in patients with SSc. The focuSSced trial did 
not meet its 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. There 
also was not a statistically significant effect on the primary endpoint 
of mRSS in the faSScinate trial. 
 
   However, in the overall population of the focuSSced study, patients 
treated with Actemra/RoActemra, 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 randomised into the focuSSced study, 68 
patients (65%) in the Actemra/RoActemra 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/RoActemra 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/RoActemra compared to placebo was 
-5.88 vs. -3.77, mean difference -2.11. 
 
   The safety profile for Actemra/RoActemra through week 48 in the 
focuSSced study was comparable for SSc-ILD and SSc patients overall, and 
in both the focuSSced and faSScinate studies was consistent with the 
known safety profile of Actemra/RoActemra. 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/RoActemra since the medicine was launched in the United States 
in 2010. 
 
   About the focuSSced Trial 
 
   The focuSSced (NCT02453256) study was a Phase III, multicentre, 
randomised, double-blind, placebo-controlled, parallel-group trial to 
assess the efficacy and safety of Actemra/RoActemra versus placebo in 
people living with systemic sclerosis. Patients were randomised in a 1:1 
ratio to receive weekly subcutaneous injections of 162 mg of 
Actemra/RoActemra or placebo during the 48-week, double-blinded, 
placebo-controlled period, followed by open-label Actemra/RoActemra 162 
mg administered subcutaneously every week for another 48 weeks. Rescue 
treatment was allowed during the treatment period after 16 weeks for 
>10% ppFVC decline or after 24 weeks for worsening skin fibrosis. 
 
   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 randomised, 68 patients (65%) in 
the Actemra/RoActemra 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/RoActemra 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/RoActemra 
 
   Actemra/RoActemra was the first approved anti-IL-6 receptor biologic, 
and is available in both intravenous (IV) and subcutaneous (SC) 
formulations for the treatment of adult patients with moderate-to-severe 
active rheumatoid arthritis (RA). Actemra/RoActemra can be used alone or 
with methotrexate (MTX) in adult RA patients who are intolerant to, or 
have failed to respond to, other disease-modifying anti-rheumatic drugs 
(DMARDs). In Europe, RoActemra IV and SC are also approved for use in 
adult patients with severe, active and progressive RA who previously 
have not been treated with MTX. Actemra/RoActemra IV and SC are also 
approved globally for polyarticular juvenile idiopathic arthritis (pJIA) 
and systemic juvenile idiopathic arthritis (sJIA) in children two years 
of age and older. Actemra/RoActemra SC is approved globally for giant 
cell arteritis (GCA), and Actemra/RoActemra IV is approved for the 
treatment of chimeric antigen receptor (CAR) T-cell-induced severe or 
life-threatening cytokine release syndrome (CRS) in people two years of 
age and older. Actemra/RoActemra was the first approved treatment for 
sJIA, GCA and CRS. Actemra SC is now approved in the U.S. for slowing 
the rate of decline in pulmonary function in adult patients with 
systemic sclerosis-associated interstitial lung disease (SSc-ILD). In 
addition to the above-mentioned indications, in Japan Actemra IV is also 
approved for the treatment of Castleman's disease and adult Still's 
disease, and the Actemra SC formulation is approved for Takayasu 
arteritis. Actemra/RoActemra is part of a co-development agreement with 
Chugai Pharmaceutical Co., Ltd and has been approved in Japan since 
April 2005. Actemra/RoActemra is approved in more than 110 countries 
worldwide. 
 
   About Roche 
 
   Roche is a global pioneer in pharmaceuticals and diagnostics focused on 
advancing science to improve people's lives. The combined strengths of 
pharmaceuticals and diagnostics under one roof have made Roche the 
leader in personalised healthcare -- a strategy that aims to fit the 
right treatment to each patient in the best way possible. 
 
   Roche is the world's largest biotech company, with truly differentiated 
medicines in oncology, immunology, infectious diseases, ophthalmology 
and diseases of the central nervous system. Roche is also the world 
leader in in vitro diagnostics and tissue-based cancer diagnostics, and 
a frontrunner in diabetes management. 
 
   Founded in 1896, Roche continues to search for better ways to prevent, 
diagnose and treat diseases and make a sustainable contribution to 
society. The company also aims to improve patient access to medical 
innovations by working with all relevant stakeholders. More than thirty 
medicines developed by Roche are included in the World Health 
Organization Model Lists of Essential Medicines, among them life-saving 
antibiotics, antimalarials and cancer medicines. Moreover, for the 
twelfth consecutive year, Roche has been recognised as one of the most 
sustainable companies in the Pharmaceuticals Industry by the Dow Jones 
Sustainability Indices (DJSI). 
 
   The Roche Group, headquartered in Basel, Switzerland, is active in over 
100 countries and in 2020 employed more than 100,000 people worldwide.

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