La Jolla Pharmaceutical, a San Diego-based developer of life-threatening diseases therapies, has received Orphan Drug designation from the FDA for two novel compounds for fibrodysplasia ossificans progressiva (FOP).
The compounds that received Orphan Drug designation are small-molecule kinase inhibitors designed to selectively block a specific member of the bone morphogenetic protein (BMP) type-I receptor family, ALK2. La Jolla recently entered a worldwide, exclusive license agreement with Vanderbilt University covering this technology. The seven members of the BMP type-I receptor family, activin receptor-like kinase (ALK) 1-7, play critical roles in human development and physiology. In turn, the improper activation of these receptor pathways is responsible for a wide range of disease conditions.
FOP is a rare genetic disorder where the body turns muscle into bone. FOP is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway. In early childhood, afflicted individuals develop soft tissue swellings that transform into bone. Development of such lesions is exacerbated by trauma, and surgical intervention leads to dramatic and explosive new bone growth. The median survival of FOP patients is about 40 years, and death often results from complications of thoracic insufficiency syndrome, which is the inability of the thorax to support normal respiration.
“There are no known treatment options available for patients suffering from FOP, and the Orphan Drug designations recognize the significant unmet need that exists within this disease,” said George F. Tidmarsh, M.D., Ph.D., president and chief executive officer of La Jolla.