The European Commission recently granted orphan drug designation to Gentium’s (GENT) Defitelio for the prevention of graft versus host disease (GvHD). This designation has entitled Defitelio to 10 years of marketing exclusivity in the EU.
Last month, the drug received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). We note that in the EU orphan drug designation is given to advance the development of drugs intended to treat, prevent or diagnose life-threatening or very serious conditions that are rare and affect not more than 5 in 10,000 people in the EU.
We remind investors that Defitelio was approved in the EU in Oct 2013 for treating and preventing hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation therapy. In Jul 2013, the EMA’s Committee for Medicinal Products for Human Use (CHMP) had recommended the approval of Defitelio for treating VOD.
Gentium has encountered hiccups in its bid to get the drug approved in the U.S. for the VOD indication. The company voluntarily withdrew its new drug application (NDA) in Aug 2011 after the U.S. Food and Drug Administration (FDA) identified several “Refuse to File” issues pertaining to the NDA. Gentium is looking to address the issues raised by FDA and subsequently resubmit the NDA.
Defitelio has been granted orphan drug status by both the FDA and EMA to treat and prevent VOD. Additionally, it also has a Fast Track Designation by the FDA for the VOD indication.
Gentium currently carries a Zacks Rank #2 (Buy). Currently, companies like Santarus, Inc. (SNTS), Jazz Pharmaceuticals Public Limited Company (JAZZ) and Actelion Ltd. (ALIOF) look better positioned with a Zacks Rank #1 (Strong Buy).
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