The initiation of the expanded access program will not impact enrolment in the company’s Phase III clinical trial as only patients who are not eligible to participate in or otherwise lack access to the Phase III clinical trial will be able to participate in the expanded access program.
Under an expanded access program, the FDA allows early access to investigational drugs that are being developed to treat serious diseases for which there is no satisfactory alternative therapy. The Company will collect additional data from the expanded access program patients to support its planned new drug application for the use of Defibrotide to treat severe veno-occlusive disease.
Laura Ferro, president and CEO of Gentium, said: “We decided to undertake this expanded access program due to the large numbers of requests for Defibrotide compassionate use investigational new drug applications.”