Children suffering from active systemic juvenile idiopathic arthritis (sJIA) will now get access to the first treatment specifically approved for the illness in the US after regulators said yes to Roche's Actemra.
The US Food and Drug Administration has expanded RoActemra/Actemra's (tocilizumab) label to include the treatment of children aged two and above with sJIA, a type of potentially life-threatening inflammatory arthritis with spiking fever, skin rash, anaemia, enlargement of the liver and/or spleen and inflammation of the lining of the heart and/or lungs.
JIA is estimated to affect around 1 to 2 per 1,000 children, with sJIA affecting about 10% of these. Of all childhood arthritis subtypes, sJIA is considered to have the worst long-term outlook for patients, with an overall mortality rate estimated to be between 2%-4%.
To date, patients were reliant on high-dose corticosteroids to help control symptoms, but not only do these fail to improve the prognosis they also come with a bag of nasty side effects, highlighting the urgency for a new and effective treatment to help better manage the condition.
The FDA's approval of Actemra in this setting is based on an international Phase III trial with 112 patients aged two-17 years old, which showed that 85% of those given Roche's drug for three months had a 30% improvement in the signs and symptoms of sJIA including an absence of fever, a key characteristic of the condition.
Despite the rarity of sJIA, clearance of Actemra's label extension is good news for Roche given that it now has access to 100% of the market, and the Swiss drugmaker will be hoping that regulators in Europe follow suit.
Actemra is already cleared in Europe for use (in combination with methotrexate) to treat moderate to severe rheumatoid arthritis in adults who have failed to responded to prior therapy, and is on the US market for adults who have not responsed to one or more TNF inhibitors.