FDA Approves Otezla to Treat Oral Ulcers Associated with Behçet’s Disease

The U.S. Food and Drug Administration has approved Otezla for the treatment of adult patients with oral ulcers associated with Behçet’s disease, a rare, inflammatory disease.

Otezla is the first and only approved treatment option for oral ulcers associated with Behçet’s disease, a rare, chronic, multisystem inflammatory disease that is difficult to treat.

Behçet’s disease causes inflammation in blood vessels throughout the body. Signs and symptoms may include mouth sores, eye inflammation, skin rashes and lesions, joint swelling, enlarged veins and arteries, inflammation in the brain and genital sores. The symptoms vary from person to person and may disappear and recur. The cause of Behçet’s disease is unknown, and it is believed that certain genes are associated with a higher risk of developing the disorder. Individuals in their twenties and thirties are most likely to develop Behçet’s disease.

“Oral ulcers are a recurring and debilitating manifestation that affects nearly everyone living with Behçet’s disease and have an important negative impact on the quality of life for these patients,” said Yusuf Yazici, clinical associate professor in the department of medicine, New York University Langone Health. “In the clinical trial, Otezla demonstrated improvements in measures of oral ulcers at week 12. Otezla has the potential to be a needed treatment option for U.S. patients and their physicians, who previously had limited options available.”

Otezla, or apremilast, is an oral, selective inhibitor of phosphodiesterase 4 (PDE4). The FDA approval was based on efficacy and safety results from the randomized, placebo-controlled, double-blind phase 3 RELIEF study evaluating Otezla in 207 adult patients with Behçet’s disease with active oral ulcers who were previously treated with at least one nonbiologic medication and were candidates for systemic therapy.

Results showed Otezla resulted in a 42.7 point reduction from baseline in the pain of oral ulcers as measured by the visual analog scale at week 12, compared with an 18.7 point reduction with placebo, with 52.9 percent of patients who received Otezla ulcer-free at week 12 compared to 22.3 percent in the placebo arm. The proportion of patients achieving oral ulcer complete response by week 6 and who remained oral ulcer-free for at least six additional weeks during the 12-week treatment phase was 29.8 percent in the Otezla arm compared to 4.9 percent in the placebo arm.

“Behçet’s Disease is a chronic inflammatory disease in which patients present with symptoms such as oral ulcers that can have a significant impact on daily life,” said Mirta Avila Santos, executive director, American Behçet’s Disease Association. “Today’s approval for Otezla marks an important milestone for people with Behçet’s Disease who have been eagerly waiting for treatment options for their oral ulcers.”

The FDA initially approved Otezla for the treatment for patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy. It is also approved for adult patients with active psoriatic arthritis.

Celgene says it anticipates a regulatory decision for Otezla in oral ulcers associated with Behçet’s disease from the Pharmaceuticals and Medical Devices Agency in Japan in the second half of 2019. The company is also seeking approval in the European Union.

Photo: Yusuf Yazici, clinical associate professor in the department of medicine, New York University Langone Health

Author: Rare Daily Staff