RARE Daily

Eton Pharmaceuticals Approved Product for Ultra-Rare

October 4, 2023

Rare Daily Staff

Eton Pharmaceuticals said it has entered into an agreement to acquire an abbreviated new drug application for nitisinone Capsules Oakrum Pharma’s Chapter 11 bankruptcy proceeding.

The transaction has been approved by the bankruptcy court and is expected to be effective on October 12, 2023. The acquired product was approved by the U.S. Food and Drug Administration in May 2023 for the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

HT-1 is a rare inborn error of metabolism caused by a lack of the enzyme fumarylacetoacetate hydrolase (FAH) which leads to accumulation of tyrosine and its metabolites in the liver. HT1 leads to cirrhosis, liver failure, hepatocellular carcinoma, and is ultimately fatal if untreated.

“With a patient population of less than 500, nitisinone is another opportunity for Eton to deliver on its mission of providing medicines to and supporting patients and families with rare conditions. Nitisinone is our fourth FDA-approved product, and further advances us toward our goal of having ten commercial rare disease products on the market by the end of 2025,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “Nitisinone also shares the same metabolic geneticist prescriber base as our carglumic acid and betaine products, so this is an attractive opportunity to leverage our existing sales force and relationships with prescribers.”

Eton expects to launch the product in the first quarter of 2024 and plans to offer its Eton Cares support program to all patients. The program is designed to help patients access treatment, providing prescription fulfillment services, insurance benefits investigation, educational support, and help in obtaining financial assistance for qualified patients, among other services.

The current nitisinone market is estimated to be more than $50 million annually, and Eton believes the company’s commercial advantages, including its Eton Cares program, existing relationships with prescribers, and experienced sales force should allow it to capture a meaningful percentage of the market.

Photo: Sean Brynjelsen, CEO of Eton Pharmaceuticals

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