FDA grants cardiotrophin-1 Orphan Drug status for acute liver failure treatment to Digna Biotech

23-Sep-2011 - Spain

Digna Biotech announced that the Food and Drug Administration (FDA) granted cardiotrophin-1 (CT-1) Orphan Drug status for use in the treatment of acute liver failure (Designation request 11-3507).

This provides more incentives to work on cures for this disease, which has an incidence of 2,000 cases per year in the United States, according to the findings reported by Acute Liver Failure Study Group (ALFSG) (Lee, Squires et al. 2008). In August, the European Medicine Agency (EMA) granted also the Orphan Drug status of cardiotrophin-1 for the treatment of acute liver failure (EU/3/11/893).

Pablo Ortiz, CEO at Digna Biotech, commented, "The experimental findings that support this new indication of CT-1 are very strong and we are confident that they can be converted in a clinical valuable effect in patients suffering from acute liver failure of different etiology. Acute liver failure is a very severe condition with a high mortality rate and very limited therapeutics possibilities that needs effective treatments. The advantage of having the product at the clinical stage starting Phase I allow us to plan Phase II for this indication by the end of next year."

Cardiotrophin-1 is a naturally occurring chemical messenger in the blood that has antioxidant (a molecule that prevents the oxidation of other molecules) and anti-inflammatory properties. Liver cells actively produce cardiotrophin-1 as part of the defense mechanism against injury. The medicine is expected to work the same way as naturally occurring cardiotrophin-1 by acting as a messenger to the damaged liver cells, helping them to recover and restoring their function. The effects of cardiotrophin-1 have been evaluated in experimental models.

Cardiotrophin-1 is co-developed for its use in organ transplantation and tissue regeneration by Digna Biotech and Biotecnol (The Consortium). Both of the companies signed an Exclusive License and Option Agreement with Genentech, Inc. (a fully owned subsidiary of the Roche Group) in September 2009. Pre-clinical and clinical development of cardiotrophin-1 was funded by a number of private and public entities in Spain: ClaveSuan, the Center for Industrial and Technological Development (CDTI), and the Government of Navarra. Only in liver resection and transplantation, cardiotrophin-1 may generate revenues of 350 million of euros per year.

https://www.bionity.com/en/news/134515/fda-grants-cardiotrophin-1-orphan-drug-status-for-acute-liver-failure-treatment-to-digna-biotech.html