Cetos

Cetos Therapeutics was a specialty biotechnology company focused on the development and commercialization of orphan drugs for rare metabolic disorders. The company is best known for its role in bringing carglumic acid (marketed as Carbaglu) to the United States market for the treatment of N-acetylglutamate synthase (NAGS) deficiency. NAGS deficiency is an ultra-rare urea cycle disorder that leads to life-threatening hyperammonemia, and Cetos successfully navigated the regulatory pathway to secure FDA approval for the therapy in 2010. Following the commercial launch and the establishment of the drug's clinical utility, the company's primary assets were acquired by Recordati (Orphan Europe). The company's work significantly improved the standard of care for patients with rare metabolic conditions who previously had limited pharmacological options.