Medivir has announced that its selective cathepsin K inhibitor, MIV-711, has been granted Orphan Drug Designation (ODD) by the FDA for the treatment of Osteogenesis Imperfecta (OI).
Orphan drug designation is granted to investigational treatments for rare diseases that affect fewer than 200,000 people in the United States.
The designation assists in developing drugs, tax credits, exemptions from FDA fees and seven years of marketing exclusivity.
"OI is a rare, genetic disorder that negatively affects the body’s ability to produce type I collagen, which leads to bone fragility, causing bone deformities and frequent fractures, often with minimal or no trauma," said Jens Lindberg, CEO of Medivir.
"There are multiple subtypes of OI, ranging from mild to severe, where the most severe types are not compatible with life and patients die shortly after birth, having suffered from multiple fractures."
"There is a significant unmet medical need, as there are no approved treatment options for patients diagnosed with OI."
"We are delighted that MIV-711 has been granted ODD by the FDA, which strengthens the evidence base for its ability to positively impact bone remodelling and its potential to treat bone-related disorders."
To gain ODD, supportive data suggesting that the drug may be effective in the disease is required.
MIV-711 has shown, in an OI-specific animal model, a significant and dose-dependent positive impact on bone strength and quality as well as bone morphology, supporting potential benefit in treating patients with OI.
Clinical data in osteoarthritis further support that MIV-711 has a positive effect in the prevention of bone degradation, translating to potential clinical benefit in OI.
MIV-711 inhibits cathepsin K, which is the main enzyme by which osteoclasts, which are involved in bone resorption and cartilage degradation, cleave collagen in the bone matrix.
Cathepsin K inhibition protects the damaged bone by reducing bone resorption and promoting bone formation, thereby addressing the key mechanisms causing pathological changes in OI, with the potential to minimise long-term negative effects.