Link to Hebrew report
Major milestone achieved as orphan status grants Enlivex seven years of exclusivity for treatment based on immune tolerance for GVHD
Jerusalem, Israel – April 3, 2013 – Hadasit Bio-Holdings Ltd. (TASE: HDST, OTC: HADSY), a publicly traded portfolio of biotech companies, all based on intellectual property developed and owned by Hadassah University Hospital, Israel’s foremost medical research center, announced today that one of its flagship portfolio companies, Enlivex Therapeutics, was awarded orphan drug status by the FDA for its ApoCell treatment. The orphan drug designation provides for seven years of exclusivity, guaranteeing no competition irrespective of patents and significant regulatory relief, which will likely shorten the marketing approval of the drug in the US and reduce costs.
The ApoCell treatment is based on inducing immune tolerance and preventing Graft-versus-Host Disease (GVHD), which affects up to 70% of heterologous bone marrow transplant recipients and can be lethal.
Enlivex CEO, Alon Moran, commented, "The results from our Phase I/II clinical trials showed safety as well as an impressive indication of efficacy using our ApoCell treatment, which reduced severe GVHD complications in heterologous bone marrow transplant recipients. We intend to pursue the development of the drug through full marketing approval in the US."
GVHD affects 30,000 people annually in the US and Europe. GVHD is an autoimmune disease in which the immune system of the new transplant attacks the patient’s organs, primarily the liver, intestines and skin. The disease can cause significant morbidity and is usually fatal if a patient reaches high grades. To date, no effective treatment has been found for this disease. The treatments that do exist are based on suppressing the immune system in order to decrease the attack on the patient's organs, but this process weakens the body and exposes it to other diseases. The ApoCell treatment, on the other hand, is based on normal biological activity and initiates a state of immune tolerance. This mechanism is naturally called into action by the body when removing cells that are in the process of programmed cell death (apoptosis). ApoCell launches this mechanism into action during the window of opportunity for treatment, close to the time of the bone marrow transplant.
"Receiving orphan drug status from the FDA is an important regulatory recognition of the drug's necessity," said Ophir Shahaf, CEO Hadasit Bio-Holdings. "The future development track of the drug is relatively short, and the company expects regulatory relief along with tax breaks as part of the approval of the drug's orphan status. This approval paves the way for the continued advancement and clinical development of the drug, and towards entering the American market that is estimated to be in the hundreds of millions of dollars annually. The exclusivity section provides assurance in protecting the product from competition, and the status as a whole instills an advantage in the regulatory process with the authorities, creating a significant competitive edge and building value for our shareholders."