The European Commission has approved AbbVie’s RINVOQ (upadacitinib) for children aged two and older with active polyarticular juvenile idiopathic arthritis (pJIA).

 

Thus, opening a new oral treatment option for patients whose disease has not responded adequately to, or who cannot tolerate, one or more disease-modifying anti-rheumatic drugs (DMARDs).

 

The approval covers polyarticular rheumatoid factor-positive and rheumatoid factor-negative disease as well as extended oligoarticular disease. RINVOQ can be used alone or in combination with methotrexate.

 

"The European Commission's approval of RINVOQ for pJIA expands the treatment options available to pediatric patients and their families, offering the potential for remission, improvements in pain and physical function," said Roopal Thakkar, executive vice president, research and development, chief scientific officer, AbbVie. 

 

"RINVOQ's new indication continues to build on our differentiated immunology portfolio with breadth and depth across therapeutic areas and patient populations."

 

The decision was supported by data from the Phase 1 SELECT-YOUTH study, along with data from adults with rheumatoid arthritis. The global, open-label study evaluated RINVOQ in patients aged 2 to under 18 with polyarticular-course juvenile idiopathic arthritis.

 

In the SELECT-YOUTH study, 66.4% of patients achieved an American College of Rheumatology pediatric 70 response at week 12. By week 48, the rate had risen to 79.5%.

 

Remission, measured using the Juvenile Arthritis Disease Activity Score based on C-reactive protein, was achieved by 23.0% of patients at week 12 and 43.4% at week 48.

 

Patients also reported improvements in physical function and pain. The mean change from baseline in Child Health Assessment Questionnaire scores was −0.46 at week 12 and −0.64 at week 48. Mean pain scores improved by −26.2 and −30.8 points at the same time points.

 

The study included 122 patients.

 

No new safety risks were identified, according to AbbVie. The rate of treatment-emergent adverse events was 499.6 events per 100 patient-years, while serious adverse events occurred at a rate of 10.2 events per 100 patient-years. Adverse events leading to discontinuation occurred at a rate of 2.7 events per 100 patient-years.

 

No major cardiovascular or thrombotic events, malignancies or deaths were reported during the study period, and no negative impact on growth or development was observed.

 

"The SELECT-YOUTH results underscore that clinical remission for children with pJIA is not a rare hope but a critical and achievable outcome that helps reduce the risk of permanent joint damage in some of our youngest, most vulnerable patients," said Gerd Horneff, head of the Department of General Pediatrics, Asklepios Clinic, Sankt Augustin, Germany. 

 

"The approval of RINVOQ in active pJIA provides a new oral systemic treatment option for patients suffering from this often-debilitating condition."