Dutch biotech firm, NorthSea Therapeutics, has seen one of its treatments for non-alcoholic steatohepatitis (NASH) given the rare paediatric disease (RPD) designation by the US Food and Drug Administration (FDA).
The company has announced that its novel drug SEFA-6179, based on Structurally Engineered Fatty Acids (SEFAs), has been granted the designation for the treatment of Intestinal Failure-Associated Liver Disease (IFALD).
NorthSea also said the fully synthetic medium chain fatty acid analogue had been shown to be well tolerated by patients in a Phase I study, with participants receiving doses up to 1,000 mg once daily for 14 days. Following a successful Phase I for SEFA-6179 earlier this year, the company is conducting a Phase IIa trial to investigate safety and tolerability. The clinical development plan regarding the paediatric population is under evaluation.
An RPD designation means the treatment is intended to treat life-threatening diseases that affect fewer than 200,000 people in the US, and for which the serious or life-threatening condition primarily affects individuals under 18 years of age.
As a result, if a New Drug Application for SEFA-6179 is approved by the FDA, the company may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed to receive for any later marketing application.
Professor Mark Puder, professor of surgery at Boston Children’s Hospital, said: “IFALD in paediatrics is a very challenging, life-threatening condition, with limited therapies.
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“We are eagerly awaiting new effective solutions for these vulnerable patients. I strongly believe that SEFA-6179 could be a candidate for treatment in paediatrics with IFALD, based on the strong pre-clinical evidence that has been generated.”
Rob de Ree, CEO of NorthSea Therapeutics, said: “Receiving RPD Designation from the FDA for SEFA-6179 highlights the urgent need to advance therapeutic options for paediatric patients facing the challenges of IFALD.
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