ALLOB Awarded Orphan Drug Designation

Bone Therapeutics’ allogeneic bone cell therapy product called ALLOB has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) as well as from the U.S. Food and Drug administration(FDA), The product is used in the treatment of the genetic bone disorder osteogenesis imperfecta—more commonly known as brittle bone disease.

The press release states:“Osteogenesis imperfecta is a rare genetic disorder that causes bone fragility, fractures and deformities. The estimated prevalence for Europe and the U.S. is 0.64 cases per 10, 000 inhabitants.” Treatments for the disability are limited. They include surgery and the use of bisphosphonates—strategies that are often associated with severe complications. At present here are no long-term solutions.

According to the press release, this orphan drug designation “opens up the possibility for Bone Therapeutics to further develop its allogeneic bone cell therapy product, ALLOB, for the treatment of osteogenesis imperfecta.” Since the underlying cause of the disorder is genetic, the allogeneic origin of the ALLOB cells could be a significant benefit.

Bone-forming cells can be administered systemically or locally at the site of fractures in order to improve structural integrity of the bone matrix by replacing the defective bone cells. The possibility exists that ALLOB could increase bone strength, decrease the risk of new fractures and accelerate fracture repair in these patients.

By obtaining orphan drug designation, Bone Therapeutics will benefit from market exclusivity in Europe for ten years and for seven years in the U.S. It has been almost ten years since the FDA granted an orphan drug designation in the field of osteogenesis imperfecta.

Company CEO Enrico Bastianelli said: “We are delighted to have received orphan drug designation for ALLOB. Currently, we have not initiated clinical trials in this field. However, the orphan drug designation gives us the opportunity to further enhance our product portfolio in the future and develop a more effective treatment that, contrary to the available treatments, targets the cause of the disease.”