UK-based rare and speciality diseases group Mereo BioPharma says its brittle bone disease drug BPS-804 has been accepted to participate in the European Medicines Agency's (EMA) Adaptive Pathways programme.
The adaptive pathways approach seeks to improve timely access for patients to new medicines, primarily in areas of high medical need, where it is difficult to collect data via traditional routes and where large clinical trials would unnecessarily expose patients who are unlikely to benefit.
The framework centres on closer consultation between multiple stakeholders together – regulators, health technology assessment (HTA) bodies, healthcare professionals and patients – to agree on a prospective plan to generate data on a medicine across its lifespan in areas of unmet medical need.
The programme uses existing regulatory channels to authorise medicines either conditionally or in a staggered manner in small, targeted patient populations most likely to benefit while additional clinical and real-world data is gathered to support wider clearance.
BPS-804 is being developed for the treatment of osteogenesis imperfecta (brittle bone disease), a rare genetic disorder characterised by fragile bones that break easily, for which there are currently no treatments approved by the EMA or US Food and Drug Administration.
Mereo's drug works by inhibiting sclerostin, which itself inhibits the activity of bone-forming cells, known as osteoblasts. The firm believes that this action will induce or increase osteoblast function and maturation, increasing bone formation and reducing bone resorption, thereby reducing bone fragility and fractures in OI patients.
"Osteogenesis imperfecta is a serious, debilitating and painful disease where we believe BPS-804 could reduce fractures and improve quality of life in OI patients. We are on track to initiate a Phase IIb trial for BPS-804 in H1 2017 and as part of the Adaptive Pathways we have the potential to bring this important therapy to patients sooner," said Mereo's chief executive Dr Denise Scots-Knight.
BPS-804 is classed an orphan drug in both Europe and the US.