SeaBeLife has been granted an Orphan Drug Designation by the European Medicines Agency (EMA) for its novel drug candidate, SBL01.
The biotech's small molecule therapeutic has been designed to block cellular necrosis, while also inhibiting ferroptosis — both of which are associated with acute liver failure.
SeaBeLife's molecule can specifically inhibit the induction cascade of these two cell death mechanisms, allowing for the protection and restoration of hepatocytes, and — therefore — liver functionality.
Acute liver failure is a life-threatening disease, which occurs without a pre-existing liver disorder. It can result in brain dysfunction, as well as a bleeding disorder, and often impacts young people.
Since the disease has such a high mortality rate and limited treatment options, SeaBeLife will now be able to benefit from regulatory and financial benefits if the drug is approved.
The company will also be able to access protocol assistance from the EMA, while also being subject to reduced fees and ten years of market exclusivity.
SeaBeLife's CEO and co-founder, Morgane Rousselot, commented: “We are thrilled to receive the orphan drug designation from European authorities. This is an important recognition of our strong preclinical research dataset; it reinforces our determination to push ahead with our approach in treating acute liver diseases and serious ophthalmologic disorders,”
“This achievement comes at a time when we are actively preparing a Series A fundraising with venture capital funds, institutional funds and family offices.”
SBL01 is a first-in-class small molecule 'dual inhibitor of both necroptosis and ferroptosis'. Regulated cell death, such as necroptosis and ferroptosis, are key processes in acute liver failure where cells degenerate abruptly. The company’s molecule is capable of specifically inhibiting the induction cascade of these two cell death mechanisms and thereby enabling cellular protection and restoration of liver function.