New FDA Drug Approvals, Indications for Rheumatic Diseases

A recent presentation at the American College of Rheumatology/Empowering Rheumatology Professionals Annual Meeting highlighted the latest information from the Food and Drug Administration (FDA) related to rheumatic diseases.

Rachel L. Glaser, MD, clinical team leader in the division of pulmonary, allergy, and rheumatology products at the FDA, gave a presentation addressing safety issues concerning recently approved rheumatic disease agents. Dr. Glaser is also a practicing rheumatologist.

Regarding new drug approvals, upadacitinib was approved for the treatment of rheumatoid arthritis (RA).

Upadacitinib (Rinvoq) is a JAK inhibitor indicated to treat adult RA patients with moderately to severely active disease who did not have an adequate response to methotrexate. The recommended dosing regimen is 15 mg/day.

“The assessment of the efficacy and safety of upadacitinib is primarily derived from five phase 3 studies, which were designed to assess once-daily upadacitinib dosing as monotherapy and as combination therapy with methotrexate or other conventional [disease-modifying antirheumatic drugs] DMARDs,” said Dr. Glaser. The studies include a diverse patient population who were either methotrexate-naïve or had an intolerance/inadequate response to methotrexate or conventional DMARDs, or had an inadequate response/intolerance to biologic DMARDs.

Across the studies, upadacitinib-treated patients “achieved higher ACR20 response rates compared to methotrexate monotherapy or placebo, respectively, at the primary efficacy endpoint,
said Dr. Glaser. “Similar trends were observed for ACR50 and ACR70 responses, as well as DAS28-CRP <2.6.”

Several existing drugs were approved for new indications, including:

  • Nintedanib (Ofev), for slowing the rate of decline in pulmonary function in SSc-ILD. This new indication was approved on September 6, 2019. The dosing regimen recommendation is 150 mg twice a day, about 12 hours apart; medication should be taken with food. (In the event of adverse reactions, a temporary dose reduction to 100 mg may be tried.)
  • Ixekizumab was approved for active ankylosing spondylitis. The recommended dosing regimen is 160 mg by way of subcutaneous injection at week zero, and then 80 mg every four weeks.
  • Apremilast (Otezla) was approved for use in adult patients with Bechet’s Disease to treat disease-related oral ulcers. The approved dosing regimen is 30 mg PO twice daily after dose titration.
  • Certolizumab pegol (Cimzia) was approved for active non-radiographic axial spondyloarthropathy. The suggested dosing regimen is an initial 400 mg dose at weeks two and four, followed by 200 mg every other week or 400 mg every four weeks.

Other existing drugs also received expanded indications:

  • Belimumab (Benlysta), previously indicated for adults with systemic lupus erythematosus (SLE), is now approved for SLE patients aged five to 17 years. The suggested dosing regimen is intravenous (IV) 10 mg/kg at two-week intervals for the first three doses and then at four-week intervals.
  • Rituximab (Rituxan) was previously approved to treat non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, RA, and pemphigus vulgaris, and is now approved as a follow-up treatment for GPA/MPA patients aged two and older. For GPA/MPA pediatric patients, the recommended dose is 375 mg/m2 once a week for four weeks. As a follow-up treatment in pediatric patients, it is recommended at two 250 mg/m2 IV infusions separated by two weeks, and then a 250 mg/m2 IV infusion months thereafter depending on clinical evaluation.