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

Digna Biotech, a Spanish biotechnological company, announces 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."

Acute liver failure is a rare disease that is manifested by a sudden loss of the normal liver functions in a patient with a previously normal liver and without evidence of chronic liver disease. The most common first sign of liver failure is yellowing of the skin (jaundice). This condition has serious complications such as bruising and bleeding due to impaired blood clotting as well as cerebral edema (swelling around the brain), convulsions (fits) and coma. The most common causes of acute liver failure are toxic damage (e.g. alcohol and drugs such as paracetamol) or viral hepatitis (an infectious disease that affects the liver). Acute liver failure is a long-term debilitating and life-threatening disease because of its damaging effects on the brain and other organs.

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.

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About cardiotrophin 1 (CT-1)

Cardiotrophin-1 is a member of the interleukin-6 cytokine family. Recently, investigators from the Centre for Applied Medical Research (CIMA), of the University of Navarra, have discovered that this protein is able to stimulate hepatic regeneration as well as to protect hepatocytes during clinical situations of acute hepatic damage. This means that CT-1 may be beneficial for those patients who undergo liver transplantation or extensive hepatic resection. CT-1 has been already granted Orphan Drug Status by the EMA for the prevention of the ischemia/reperfusion injury associated with solid organ transplantation (EU/3/06/396) and it is also obtained FDA Orphan Drug Status for protecting the liver from ischemia/reperfusion injury inherent to the procedure of transplantation (designation request 07-2449). In addition, CT-1 has recently been shown to prevent graft injury and inflammatory response and prolong survival in animal kidney transplant model, and acute liver failure. Promising results for the treatment of obesity and metabolic syndrome has been also found.

About Digna Biotech

Digna Biotech, a biotechnological company, is a spin-off of the University of Navarra (Pamplona, Spain) and is funded by a group of investors representing some of Spain's major corporations and financial institutions. It is committed to develop new drugs based on the IP generated by the Center for Applied Medical Research (CIMA) and to forge partnerships with pharmaceutical industry to ensure complete clinical development and commercial launch. Its extensive pipeline reflects the four major avenues of research pursued by CIMA: Cardiovascular, Gene Therapy, Hepatology and Oncology. Inspired by similar university-industry collaboration projects in USA, Digna Biotech presents a pioneering business model in Spain. In cooperation with CIMA, CIFA (Center For Applied Pharmacobiology Research), a number of Schools of the University of Navarra (especially the School of Pharmacy and its Galenic Department), and the Clínica Universidad de Navarra, Digna Biotech is carrying out a range of activities to ensure the clinical trial development of the products. Digna Biotech has to acknowledge the supportive work made by Idifarma during the whole process of the orphan procedures.

As a direct result of the company's commitment to translational research (bench to bedside) and thanks to the effort made by the staff of Digna Biotech and CIMA, three products of its pipeline are now in the clinical phase. The company plans to enter a new product every year in clinical trials to reach the patient with innovative products for unmet medical needs.

More information: http://www.dignabiotech.com and www.cima.es

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