Orphan Drug Designation granted to Versantis

The Zurich based Versantis founded in 2015 is a preclinical stage pharmaceutical company focused on rare liver diseases. It is committed to the development of life-saving therapies for acute liver failure patients for which the current available care is not satisfactory. As its first product, Versantis developed a lead product candidate “VS-01” which is considered the first safe and effective treatment for acute-on-chronic liver failure (ACLF), a life-threatening condition of high unmet medical need.

In May 2016, the Committee for Orphan Medicinal Products (COMP) issued a positive opinion on orphan medicinal product designation for VS-01 for the treatment of acute liver failure, a condition in which the rapid deterioration of liver function results in hepatic encephalopathy, coagulopathy and even progressive multi organic failure in the absence of a pre-existing liver disease.

To qualify for orphan designation, a medicine must be intended for the treatment of a disease that is life-threatening, the prevalence of the condition in the EU must not be more than 5 in 10,000, no satisfactory method of treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition. In light of the overall data submitted and the discussions within the committee, the COMP judged that the startup submitted sufficient data to support the medical plausibility of VS-01 for the purpose of designation.

Versantis will as a result access a wide range of sponsor incentives from the Committee to further develop its VS-01 including; protocol assistance during clinical development, access to the centralised authorisation procedure, access to conditional approval,  market exclusivity post approval of 10 years, reduced fees for regulatory activities including protocol assistance, marketing-authorisation applications, inspections before authorisation and applications for changes to marketing authorisations made after approval, and reduced annual fees and available funding from the European Commission.

(RAN)