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Apremilast: Celgene’s Orphan Drug Phase II Results For Behçet’s Disease

June 14, 2013

Celgene’s orphan drug Apremilast is an oral agent that is currently under clinical development for the treatment of inflammatory autoimmune disorders, such as psoriatic arthritis, psoriasis, rheumatoid arthritis, and Behçet’s Disease (BD). Apremilast is currently in Phase II clinical trials for BD and in Phase III clinical trials for moderate-to-severe psoriasis and psoriatic arthritis. In January 2013, Apremilast receives FDA Orphan Drug Designation (ODD) for the treatment of BD.

BD is a rare chronic inflammatory disorder that can lead to recurrent oral and genital ulcers, skin ad eye lesions, and joint inflammation, as well as involvement of the brain and gastrointestinal tract. Prevalence of BD is highest in the Eastern Mediterranean, the Middle East, and East Asia. BD is classified as a rare or “orphan” disease by the NIH in the US and by EURODIS in Europe. It is estimated that across the high prevalence countries, there are approximately 250,000 cases of BD. There are currently no approved therapies in the US or Europe for BD.

On June 14, 2013, Celgene presents results, from a Phase II randomized, placebo-controlled trial in patients with BD, at the European Congress of Rheumatology’s (EULAR) Annual Meeting in Madrid, Spain. The Abstract presented at EULAR is available online :

FRI0331 – Apremilast for the Treatment of BEHÇET’S SYNDROME: A Phase II Randomized, Placebo-Controlled, Double-Blind Study.

Apremilast achieves statistical significance for both primary and secondary endpoints in the Phase II trial for BD :

“ … statistical significance for the primary endpoint of the mean number of oral ulcers at week 12 (APR 30 mg BID, 0.5; PBO, 2.1; p<0.0001) … The complete response rate (oral ulcer free) at week 12 was also statistically significant (APR, 71%; PBO, 29%; p<0.0001). Statistically significant and clinically meaningful responses were demonstrated across all secondary endpoints, including all patient-reported outcomes. Notably, improvement in oral ulcer pain (VAS) was significantly higher with Apremilast than with placebo (-44.7 vs -16.0; p<0.0001). Among the patients with genital ulcers at baseline (n=16), all 10/10 receiving APR had a complete response at week 12 vs 50% (3/6) receiving PBO (p=0.04).”   

Behcet’s Disease Facts

– An autoimmune disease resulting from damage to blood vessels

– Develops usually in those who are in their 20’s and 30’s

– Hard to diagnosis

– Currently no cure, and treatments are not always effective

– US prevalence rate is estimated at 1/20,000

– Prevalence rate in other countries is 1/10,000.


American Behcet’s Disease Association (ABDA)

Behcet’s Syndrome Society in the UK.

Please Note: “Spinning Device To Mix Tissue Culture Medium” by National Cancer Institute [Public domain] | Wikimedia Commons.

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