Corbus’ Scleroderma Therapy Granted Orphan Drug Designation

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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A investigational drug being developed by Corbus Pharmaceuticals Holding, Inc. to treat systemic sclerosis was granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The drug that received the status, resunab, is Corbus’ lead drug candidate, as the company is focused on developing new treatments to address rare, chronic, and severe inflammatory and fibrotic conditions.

Resunab is a novel synthetic oral drug with anti-inflammatory and anti-fibrotic proprieties, expected to treat chronic conditions, such as systemic sclerosis, or scleroderma, among others. By receiving the FDA orphan drug designation, Corbus may receive seven years of marketing exclusivity following the approval of resunab, as well as other incentives, such as federal grants, tax credits or reduced fees.

The FDA awards the orphan drug designation to promising drug treatments or biologic products expected to effectively prevent, diagnose or treat rare conditions that affects less than 200,000 patients in the United States. Resunab was already studied in pre-clinical and phase 1 studies that revealed its safety, tolerability and pharmacokinetic profile, and the FDA had already approved Corbus’ investigational new drug application (“IND”) for systemic sclerosis.

“We are very pleased to receive FDA Orphan Drug Designation for Resunab in systemic sclerosis,” stated in a press release the company’s CEO and director Yuval Cohen, PhD. “This is an important regulatory milestone for the company and a significant step forward in our clinical development of Resunab targeting this rare disease associated with such a critical unmet need for safe and effective therapeutics.”

“Based on its novel mechanism of action of triggering the inflammatory resolution pathway, we believe Resunab has the potential to become an important therapy for systemic sclerosis patients as well as other diseases in which chronic inflammation and fibrosis persist,” added Cohen. The company expects Resunab to become an effective treatment for systemic sclerosis and bring new hope for SSc patients with unmet medical needs.

There is currently no drug treatments approved by the FDA to treat systemic sclerosis, which is a chronic and severe inflammatory condition that causes fibrosis of both the skin and internal organs, becoming life-threatening as it progresses. The disease affects mainly women in mid-life and is associated with high morbidity and mortality rates.

Last March, Yuval Cohen spoke to Scleroderma News in an exclusive interview, explaining implications of developing resunab and bring it to the markets to  treat diffuse cutaneous systemic sclerosis, the most severe form of scleroderma. The company had just been cleared by the FDA to initiate its phase 2 clinical trials, which was planned to include 36 patients and monitor adverse side effects resunab treatment.