EU approval for Novartis’ rare disease drug

Global pharma company, Novartis, has received EU approval for its rare disease drug, Ilaris.

Ilaris (canakinumab) is the first interleukin-1 beta inhibitor for the treatment of active Systemic Juvenile Idiopathic Arthritis (SJIA), which is a rare and disabling form of childhood arthritis with limited treatment options. It is also the only approved treatment for SJIA that is given as a monthly subcutaneous injection.

The European Commission has approved Ilaris for use in patients aged two years and older who suffer from SJIA and have responded inadequately to previous therapy with non-steroidal anti-inflammatory drugs (NSAIDs) and systemic corticosteroids.

"The EU approval of Ilaris provides patients suffering from SJIA with a convenient new treatment option offering a favorable benefit-risk profile, administered as a single monthly subcutaneous injection. This represents another significant milestone in the development of Ilaris as a novel therapy for patients with rare, inflammatory diseases, where interleukin-1 beta plays a key role."

Timothy Wright, MD, Global Head of Development, Novartis Pharmaceuticals.

In phase 3 clinical trials, 84% of Ilaris-treated SJIA patients achieved significant improvement of systemic and arthritic symptoms after a single subcutaneous dose.

Novartis has already received approval for Ilaris as a treatment of SJIA in the US and also for the symptomatic treatment of refractory acute gouty arthritis in the EU. The drug is also approved in more than 60 countries, including in the EU, US, Switzerland and Japan for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), a rare, lifelong, genetic disorder with debilitating symptoms.