Biostage, Inc. a biotechnology company developing bioengineered organ implants to treat cancers and other life-threatening conditions of the esophagus, bronchus and trachea, announced that its Cellspan™ Esophageal Implant was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) to restore the structure and function of the esophagus subsequent to esophageal damage due to cancer, injury or congenital abnormalities.
The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition upon marketing approval, as well as certain incentives, including tax credits and a waiver of PDUFA filing fees.
"The Orphan Drug Designation for our Cellspan Esophageal Implant is an important achievement in our regulatory strategy as we advance this novel product candidate that we believe, has the opportunity to create a significant treatment paradigm shift in regenerative medicine," said Jim McGorry, CEO of Biostage. "Based on preclinical, large-animal studies conducted to date, we believe our Cellspan esophageal implants may have the potential to offer improved outcomes for patients by reducing post-operative complications and providing an improved quality of life, by prompting regeneration of the patient's own esophagus."
The Company recently provided a regulatory update of its Cellspan esophageal implant following its substantive pre-IND meeting with the FDA. The feedback provided by the FDA to Biostage's proposed clinical protocol for its esophageal implant provided clarity for a clinical path forward for its intended Investigational New Drug (IND) application and the advancement into Phase 1 human clinical studies. The Company expects to file its IND in the third quarter of 2017 and commence its Phase 1 first-in-human clinical study in the fourth quarter of 2017.
"We believe this Orphan Drug Designation and the regulatory guidance we recently received from the FDA, provide a clear development pathway which may potentially enable us to offer a promising treatment outcome for patients suffering from esophageal cancer. Our focus is steadfast on emerging as rapidly as possible as a clinical-stage company," concluded Mr. McGorry.
