FDA Rheumatology Update: New Drug Approvals, Plus Expanded Drug Indications & Safety Concerns

Drugs with new indications approved in 2019 include:

• Nintedanib (Ofev), an oral, small-molecule, tyrosine kinase inhibitor, was approved in September for slowing the rate of decline in pulmonary function in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD);
• Ixekizumab (Taltz), an interleukin-17-A (IL-17A) inhibitor, was approved in August for the treatment of active ankylosing spondylitis;
• Apremilast (Otezla), a phosphodiesterase-4 inhibitor, was approved in July for adults with oral ulcers associated with Behçet’s disease; and
• Certolizumab pegol (Cimzia), a tumor necrosis factor (TNF) inhibitor, was approved in March for the treatment of active, non-radiographic axial spondyloarthritis with objective signs of inflammation.

In 2019, the FDA also approved expanded pediatric indications for two medications:

• Intravenous belimumab (Benlysta), a BLyS-specific inhibitor, was approved for pediatric patients with systemic lupus erythematosus (SLE) from ages 5–17 years; and
• Rituximab (Rituxan, MabThera), a CD20-directed cytolytic antibody, was approved for pediatric patients with granulomatosis with polyangiitis (GPA)/microscopic polyangiitis (MPA) ages 2 and older, and as follow-up treatment for both adults and pediatric patients with GPA/MPA.

From left: Nadia Habal, Rachel Glaser & Nikolay Nikolov

Since the 2018 ACR/ARHP Annual Meeting, the FDA has approved four treatments that are biosimilar to drugs often prescribed by rheumatologists: adalimumab-adaz (Hyrimoz), adalimumab-bwwd (Hadlima), etanercept-ykro (Eticovo) and rituximab-pvvr (Ruxience). The FDA also approved several first-time generic drugs that may be prescribed for patients with rheumatic diseases, including nabumetone, febuxostat, pregabalin, tramadol hydrochloride 100 mg tablets, sildenafil oral suspension, penicillamine and bosentan, Dr. Glaser noted.

Additionally, colchicine oral solution (Gloperba), a new formulation of this drug, was approved in January 2020 for prophylaxis of gout flares in adults, providing patients with a new option for gout flare management.

“The last year has been an exciting year for the division, community, and patients. We have approved multiple products for rheumatic diseases, and approved drugs for new indications and populations, including first approvals to slow pulmonary function decline in SSc-ILD, for oral ulcers in Behçet’s disease, non-radiographic axial spondyloarthritis, pediatric patients with SLE and pediatric patients with GPA/MPA,” said Dr. Glaser.

Safety Issues
In 2019, the FDA added warnings for several important safety issues, and announced drug recalls and drug shortages, said Nadia Habal, MD, medical officer, DPARP.

Febuxostat (Uloric), a selective xanthine oxidase inhibitor, received a revised indication for chronic management of hyperuricemia in gout patients who have an inadequate response to a maximally titrated dose of allopurinol, who are intolerant to allopurinol or for whom treatment with allopurinol is not advisable.