FDA Grants Fast Track Designation to Bullous Pemphigoid Treatment, Bertilimumab

The US Food and Drug Administration (FDA) has granted a fast track designation to Immune Pharmaceuticals, Inc.’s bertilimumab for the treatment of bullous pemphigoid, a rare skin condition which causes large, fluid-filled blisters.

“I am truly grateful that the FDA has granted fast track designation to bertilimumab for the treatment of bullous pemphigoid,” shared Immune’s interim chief executive officer, Tony Fiorino, MD, PhD, in a recent statement. “There is no doubt that bertilimumab development will benefit from the opportunity to have more frequent contact with the FDA, particularly now, as we move forward with a new manufacturing process and plan for a phase 2/3 pivotal study in bullous pemphigoid.”

Previous to this decision, an open-label, investigators conducted a proof-of-concept phase 2 trial evaluating the safety and effectiveness of bertilimumab in adult patients newly diagnosed with moderate to extensive bullous pemphigoid. Eleven participants were enrolled in the trial, which consisted of 3 periods: a 2-week screening period, a 4-week open-label treatment period, and a 13-week follow-up period to evaluate the safety and efficacy of the treatment.

For the treatment portion of the trial, participants received bertilimumab intravenously on days 0, 14, and 28. They also received concomitant modest doses of prednisone that were aggressively tapered.

Patients experienced a decline in the Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score of 81% (p=0.015) from a mean baseline score of 67 at day 84 of the trial with 86% of participants experiencing 50% improvement in their BPDAI Activity Score and 57% experiencing a 90% improvement. Participants also experienced improvements in pruritus and quality of life. Due to the positive data yielded by the phase 2 trial, plans for a phase 2/3 trial is being discussed to serve as a registrational study for the rare disease.

Bertilimumab is a fully human monoclonal antibody that targets and lowers levels of eotaxin-1, a chemokine that attracts eosinophils to the site of inflammation. Bertilimumab may prevent the migration of eosinophils and other cells into inflamed tissues by neutralizing eotaxin-1 and assist in relieving associated conditions as a result.

Previously, bertilimumab was granted an orphan drug designation by the FDA.