RARE Daily

FDA Grants Rare Pediatric Disease Designation to NorthSea’s Treatment for Intestinal Failure-Associated Liver Disease

October 17, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to NorthSea Therapeutics experimental therapy for the treatment of intestinal failure-associated liver disease, or IFALD.

IFALD, also known as parental nutrition-associated liver disease (PNALD), is an orphan liver disease which is a frequent consequence of total parenteral nutrition, or TPN, a life-saving therapy for individuals with intestinal failure caused by insufficient bowel-length or function. No approved drug therapy exists for these patients.

NorthSea’s candidate, SEFA-6179, is a novel, oral, fully synthetic medium chain fatty acid analog for the treatment of IFALD. The molecule has been shown to be well tolerated in a phase 1 study in healthy volunteers.

The granting of Rare Pediatric Disease designation for SEFA-6179 underscores the critical need for novel therapies to address IFALD in the pediatric population. There is also an unmet need in adults where prolonged Total Parenteral Nutrition (TPN) use can induce IFALD with slightly different clinical manifestations versus pediatrics. Having successfully completed phase 1 for SEFA-6179 earlier this year, NorthSea is currently conducting a phase 2a trial to investigate pharmacokinetics, safety/tolerability, as well as pharmacodynamics effects in IFALD patients. The clinical development plan in pediatrics is under evaluation.

FDA’s Rare Pediatric Disease designation is intended to encourage the development of new drugs and biologics for the treatment of rare pediatric diseases, which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. Under this program, if a New Drug application for SEFA-6179 for the treatment of IFALD is approved by the FDA, NorthSea may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred to a third party. Most recently, Sarepta Therapeutics sold a voucher for $102 million.

“Receiving RPD Designation from the FDA for SEFA-6179 highlights the urgent need to advance therapeutic options for pediatric patients facing the challenges of IFALD,” said Rob de Ree, CEO of NorthSea Therapeutics.

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