FDA Approves Apremilast for Behcet's Oral Ulcers Save

The US Food and Drug Administration (FDA) has approved apremilast (Otezla) for the treatment of oral ulcers associated with Behçet’s Disease, a rare chronic multisystem inflammatory disease that affects about 5 in 100,000 persons in the USA.

Apremilast, an oral phosphodiesterase 4 inhibitor was previously FDA approved for use in psoriasis and psoriatic arthritis where it has been used in over 250,000 patients in the U.S., since its FDA approval in 2014.

The approved dose for Behcet's disease will be 30 mg twice-daily.

FDA approval on Friday, July 19th, was based on efficacy and safety seen in a Phase 3 study that included 207 Behçet’s disease patients with active oral ulcers who were randomized to apremilast (APR) or placebo (PBO). This 12 week study showed significant benefits of APR compared with PBO:

• Less oral ulcer pain (-42.7 vs -18.7 points respectively)
• More were oral ulcer-free at week 12 (52.9% vs. 22.3%)
• Lower daily average number of oral ulcers (1.5 vs. 2.6)

Common adverse events observed with APR in this trial were diarrhea (41%), nausea (19%), headache (14%) and upper respiratory tract infection (11%).