Bone Therapeutics granted orphan drug designation by FDA and EMA for Allob in brittle bones

Belgian pharma company Bone Therapeutics (Euronext Brussels: BOTHE) has been granted orphan drug designation by the European Medicines Agency and the US Food and Drug Administration for Allob, its allogenic bone cell therapy product.

The designation has been granted for Allob’s use in brittle bone disease, a rare genetic disorder that causes bone fragility, fractures and deformities. It opens up the possibility for Bone Therapeutics to develop Allob to treat brittle bone disease, where bone-forming cells can be administered systemically or locally at the site of the fractures to improve the structural integrity of the bone matrix by replacing the defective bone cells.

Allob is the first product to be granted orphan drug designation for brittle bone disease in Europe, and it has been nearly a decade since the FDA granted the designation in this indication. Bone Therapeutics will benefit from incentives such as market exclusivity in Europe for 10 years and in the USA for seven years, once the medicine is approved for commercialization. It may also benefit from significant fee reductions in terms of protocol development, scientific advice and product registration procedures.