SAN DIEGO, CA--(Marketwired - August 05, 2013) - Vital Therapies®, Inc., a biopharmaceutical company focused on developing a cell-based therapy targeting the treatment of acute liver failure, today announced that the European Commission granted Orphan Medicinal Product Designation for C3A cells for the treatment of acute liver failure. These cells, which the company grows from its proprietary cell bank, serve as the active ingredient in ELAD®, the company's investigational artificial liver therapy.
Under EMA guidelines, Orphan Designation provides 10 years of potential market exclusivity if the designated product candidate is approved for marketing in the European Union and the designation is maintained. Orphan status also permits EMA assistance in optimizing the candidate's clinical development through participation in designing clinical protocols and preparing the marketing application. Finally, a drug candidate designated by the EMA as an Orphan Medicinal Product qualifies for a reduction in fees associated with the filing of a market authorization application and other regulatory fees associated with obtaining regulatory approval.
Vital Therapies has already received Orphan Designation in the United States for C3A cells in the treatment of acute liver failure. As such, if the company receives marketing approval for ELAD in this indication, the FDA could not approve another therapy whose active ingredient includes C3A cells for the treatment of acute liver failure, except in very limited circumstances, for up to seven years.
In other news, Vital Therapies, Inc. completed the closing of a financing tranche earlier this summer. This financing represents the third scheduled tranche of a broader financing initially announced in September of 2012. The total amount raised under this financing exceeds $60 million, of which $31 million was raised in June of 2013.
About Vital Therapies, Inc.
Vital Therapies, Inc. is a biopharmaceutical company based in San Diego, California. The company is developing ELAD, an investigational extracorporeal bio-artificial liver therapy which has been studied in six clinical trials in US, EU and Asia. The company currently has the first of three planned phase 3 clinical trials underway, with the remaining two clinical trials anticipated to begin enrolling subjects later in 2013.
ELAD is an allogeneic cellular therapy system based on our human liver-derived cells combined with a reusable bedside unit. We have customized the now-publicly available C3A cell line to create an optimized bank of cells for use in ELAD which we culture and expand through proprietary techniques. These cells have been shown to retain many key synthetic and metabolic processes of normal human hepatocytes, the primary functional cell of the liver. During ELAD therapy, blood plasma is drawn from the patient via our reusable bedside unit into the ELAD cartridges, which collectively contain 440 grams, or approximately one pound of C3A cells. The patient's blood plasma is treated by our C3A cells in a single session of continuous therapy lasting between three and ten days. We believe that ELAD therapy facilitates the recovery of liver function and has the potential to increase survival.
Vital Therapies® and ELAD® are trademarks of Vital Therapies, Inc.
This press release may contain forward-looking statements, including but not limited to, statements regarding our phase 3 clinical trials, anticipated potential benefits of the ELAD system, and expected advantages of orphan drug designations in Europe and the United States. These statements relate to future events and are subject to risks, uncertainties and assumptions about Vital Therapies, Inc., including the risks that our clinical trials may not be successful and that we may fail to receive marketing approval for ELAD in the jurisdiction. These statements are only predictions based on our current expectations and projections about future events. You should not place undue reliance on these statements. Actual events or results may differ materially. Many factors may cause our actual results to differ materially from any forward-looking statement. We do not undertake to update any forward-looking statements.