Gentium S.p.A. announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recently rendered a negative opinion on its pipeline candidate defibrotide.
Gentium is looking to get the candidate approved for treating and preventing hepatic veno-occlusive disease (VOD) in adults and children. The condition appears in patients undergoing hematopoietic stem cell transplantation therapy. VOD refers to a rare but serious deficiency. The disorder results in damage to the small blood vessels in the liver due to high doses of chemotherapy and radiation received by the patient before undergoing a stem cell transplant. We note that currently there are no approved therapies to treat VOD.
Gentium intends to appeal against the negative opinion issued by the CHMP and request the body to its adverse stance. The company believes that the benefits associated with the candidate outweigh the risks involved.
Moreover, defibrotide was recommended by the European Group for Blood and Marrow Transplantation (EBMT)as the sole option to treat VOD patients. EBMT is the largest professional body in the haematopoietic stem cell transplantation space. The body, which boasts of more than 4,200 members in 570 transplant centers in 57 countries across the globe, has expressed concern over the negative opinion issued by the CHMP regarding the approval of defibrotide in Europe.
According to the procedure laid down by the EMA regarding appeal, Gentium has to request for a re-examination of the CHMP opinion within 15 days of its receipt of the same. Moreover, Gentium can submit the grounds for appeal within 60 days.
The CHMP can review its initial opinion within a further 60 days. Under the EMA appeal procedure, Gentium has to request for re-examination of the CHMP’s opinion on the basis of the initial data submitted. The company can submit a fresh application in the EU in the event of its appeal being unsuccessful.
Gentium carries a Zacks Rank #3 (Hold). Athersys, Inc. (ATHX - Snapshot Report) , Cytokinetics, Inc. (CYTK - Snapshot Report) and Osiris Therapeutics, Inc. (OSIR - Analyst Report) look more attractive in the pharma space. All three stocks carry a Zacks Rank #1 (Strong Buy).