BioMimetic Therapeutics, Inc (FRANKLIN, Tennessee), an emerging leader in developing and commercializing bioactive drug-device combination drugs to heal musculoskeletal injuries and disease, including periodontal, orthopaedic, spine and sports injuries, announced that it has received orphan drug designation from the US FDA for its protein therapeutic, recombinant human platelet-derived growth factor (rhPDGF-BB). The therapy stimulates bone and tissue healing, for the treatment of osteonecrosis of the jaw (ONJ) which, according to the company, is a growing problem in US patients who receive high doses of bisphosphonates for the treatment of severe osteoporosis and other diseases.

Orphan drug designation was designed by the FDA to encourage the development of therapeutic products for clinical indications that affect fewer than 200,000 individuals in the US. Drugs that receive orphan drug designation obtain 7 years of marketing exclusivity from the date of drug approval as well as tax credits for clinical investigation costs, marketing application filing fee waivers, and assistance from the FDA in the drug development process.

ONJ is also known as avascular or aseptic necrosis of the mandible/maxilla, bisphosphonate-associated osteonecrosis of the jaw (BON), jaw rot, dead jaw disease, or bisphossy jaw. The typical clinical presentation of ONJ includes pain, soft-tissue swelling and infection, loosening of teeth, drainage, and exposed bone. These symptoms may occur spontaneously, or more commonly at the site of previous trauma, such as a tooth extraction. Patients may also present with feelings of numbness, and heaviness and/or tingling of the jaw, which may be diagnosed after finding exposed bone in the jaw that doesn't heal following use of current therapies.

Chemotaxic and mitogenic rhPDGF-BB stimulate a broad spectrum of cellular events critical for the initiation and progression of healing of musculoskeletal tissues including bones, tendons, ligaments, and cartilage.

BioMimetic received marketing approval from the FDA for its first product, GEM 21S®, a grafting material for bone and periodontal regeneration. Additionally, the company has ongoing clinical trials with its product candidate GEM OS1^TM in multiple orthopaedic bone-healing indications, including the treatment of foot and ankle fusions and the stimulation of healing of fractures of the arm. The company's product and product candidates all combine recombinant protein therapeutics with tissue-specific scaffolds to actively stimulate tissue healing and regeneration.

—A. Techman

E-mail any comments to .