Mereo BioPharma Group announced that its drug BPS-804 has been granted orphan drug designation by the European Commission (EC) in the treatment of osteogenesis imperfecta (OI).
Osteogenesis Imperfecta (OI) is a rare, chronic genetic disorder that results in bones that can break easily. The Company intends to initiate a potential registration trial of BPS-804 in the second half of 2016.
In March 2016, BPS-804 was granted orphan drug designation for the treatment of OI by the U.S. Food and Drug Administration (FDA).
OI is a rare condition that is estimated to affect a minimum of approximately 20,000 and possibly as many as 50,000 patients in the United States. In Europe, approximately 7.5 out of 100,000 people have the condition.
Current treatment largely relies on the acute management of fractures as they occur and the use of bisphosphonate drugs, although the Company believes there is no clear data demonstrating that bisphosphonate drugs reduce fractures. BPS-804 aims to demonstrate a benefit compared to placebo in terms of fractures in OI patients.
BPS-804 works by inhibiting sclerostin, which inhibits the activity of bone-forming cells, known as osteoblasts. The Company believes that by blocking sclerostin, BPS-804 will induce or increase osteoblast function and maturation of these cells, increasing bone formation and reducing bone resorption, thereby reducing fractures in OI patients. Phase 2 proof of concept data in OI patients demonstrated a statistically significant improvement in bone biomarkers and bone mineral density.
Mereo CEO Dr Denise Scots-Knight said: "Osteogenesis imperfecta is a debilitating and painful disease. The EC orphan drug designation grant follows the recent grant from the FDA.
"It is another important regulatory milestone and marks a significant step forward for BPS-804. We believe BPS-804 has the potential to become the first therapy to reduce fractures and improve quality of life in osteogenesis imperfecta patients.
"Initial clinical data generated with BPS-804 has been highly encouraging and we look forward to progressing this product candidate towards registration."
The EC may grant orphan drug designation to drugs intended for the treatment of life threatening or chronically debilitating rare diseases where no therapeutic options are either authorised or where the drugs will be of significant benefit to those affected by the condition. Rare diseases are those defined as having a prevalence of no more than five in 10,000 persons in Europe.
The designation provides development and commercial incentives, including 10 years of market exclusivity, protocol assistance on the development of the drug, including clinical studies, and certain exemptions from or reductions in regulatory fees.