The HART-Trachea won orphan drug designation for repair or replacement of tracheas that have been severely damaged by trauma or cancer, giving the company the green light to proceed with clinical trials in pursuit of market approval.
"We greatly appreciate the FDA’s support of our efforts to develop our first product, the HART-Trachea, to help patients who require a replacement trachea," HART president & CEO David Green said in prepared remarks. "Orphan designation is the 1st step toward clinical trials whose successful completion will be necessary to obtain FDA approval for use of the HART-Trachea in the U.S."
Orphan designation applies only to products intended to treat a condition that affects fewer than 200,000 people in the U.S. Since the potential patient population is so small, the burden of proof is lightened for these products, but companies must nonetheless demonstrate that the products have a reasonable likelihood of effectiveness.
If the HART-Trachea wins FDA approval, the company will enjoy a 7-year solo run on the market plus a pathway to apply for research funding, tax credits and an FDA application fee waiver, HART said.
HART spun out of Harvard Bioscience (NSDQ:HBIO) in November, taking with it all rights to the synthetic trachea regeneration technology. The companies announced late last year that the world’s first tissue-engineered artificial trachea transplant patient was "living normally without any complications or rejection of the implanted airway" 5 years after receiving the organ.