FRANCE—The European Medicines Agency (EMA) has granted SeaBeLife, a biotechnology company focused on developing drug candidates to block cellular necrosis, Orphan Drug Designation (ODD) for its drug candidate SBL01, intended for the treatment of acute liver failure (ALF).
Acute liver failure is a life-threatening condition characterized by the sudden loss of liver function in individuals without pre-existing liver disorders. This rapid deterioration leads to severe complications such as encephalopathy (brain dysfunction) and coagulopathy (bleeding disorder).
ALF is particularly concerning as it often affects young individuals and is associated with a high morbidity and mortality rate, reaching 85% when transplantation is not feasible.
In the European Union, the prevalence of ALF is estimated to be between 1.3 and 3.1 per 10,000 people, meeting the EMA’s orphan designation criteria, which applies to conditions affecting no more than 5 in 10,000 people.
With the ODD, SeaBeLife stands to benefit from various regulatory and financial incentives, including protocol assistance, reduced fees, and ten years of market exclusivity upon approval.
According to the EMA’s Committee for Orphan Medicinal Products, SeaBeLife presented non-clinical data demonstrating that SBL01, when combined with an already authorized product, significantly reduced hepatotoxicity and improved survival rates in a model of acute liver failure.
The committee recognized this as a clinically relevant advantage over the currently authorized treatment.
Expressing his gratitude for the orphan drug designation, Morgane Rousselot, CEO and co-founder of SeaBeLife, emphasized that this recognition validates their robust preclinical research.
He stated that it strengthens their resolve to continue their innovative approach in treating acute liver diseases and serious ophthalmologic disorders.
Rousselot also mentioned that this achievement aligns with their ongoing preparations for a Series A fundraising round involving venture capital funds, institutional investors, and family offices.
SBL01 is a first-in-class small molecule described as a ‘dual inhibitor of both necroptosis and ferroptosis.’
These regulated cell death processes are critical in the progression of acute liver failure, where cells degenerate rapidly.
By specifically inhibiting the initiation of these two mechanisms, SBL01 offers potential cellular protection and restoration of liver function.
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