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Doxorubicin Transdrug Granted Orphan Drug Status for Hepatocellular Carcinoma in Europe

PARIS--(BUSINESS WIRE)--Nov. 16, 2004-- BioAlliance Pharma, a biopharmaceutical company focused on the field of drug resistance, today announced that the European Commission has granted the company an 'Orphan Medicinal Product' designation for doxorubicin Transdrug(R) as a treatment for hepatocellular carcinoma (HCC), primary liver cancer. The decision follows the favorable opinion of the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). Doxorubicin Transdrug(R) is a proprietary nanoparticle formulation currently being evaluated in a Phase I/II trial in HCC at seven clinical sites in France.

The company will also file an application this month with the U.S. Food and Drug Administration to obtain orphan drug designation in the U.S.

The EMEA's 'orphan' designation promotes development of drugs to treat rare diseases or conditions, which would otherwise be unprofitable to pursue. The actual definition of an orphan drug within the EU is one for a disease that affects fewer than five people in every 10,000. The designation provides EU market exclusivity, for a particular indication, against drugs with the same principal molecular structural features and which act via the same mechanism. The market exclusivity is for a 10-year period if the sponsor complies with certain EMEA specifications. The EMEA represents 25 EU countries, including France, Germany, Italy, Spain and the United Kingdom.

In addition to marketing exclusivity, designation as an orphan drug provides other incentives including EMEA protocol assistance to optimize drug development in preparing a dossier that will meet regulatory requirements; facilitating access to the Centralized Procedure for the application for marketing approval; complete or partial waiver of fees associated with applying for marketing approval and protocol assistance; and, access to EU research funding for rare diseases.

"The orphan drug designation in the EU signifies another important step for BioAlliance in the development of Doxorubicin-Transdrug," said Dominique Costantini, M.D., president and CEO of BioAlliance Pharma. "We appreciate the EMEA's recognition of our efforts and investment in developing doxorubicin Transdrug(R) for the potential benefit of patients with this aggressive form of cancer."

About Transdrug Nanoparticle Drug Delivery
BioAlliance has developed a proprietary nanoparticle technology using PIHCA (poly-iso-hexyl-cyanoacrylate), a proprietary polymer to formulate a number of anti-cancer drugs. In the human body, these drug-loaded nanoparticles are translocated into the cancer cell where they elicit their known anti-cancer activity. Hence the name of the drug delivery technology: Transdrug(R).

The rationale for testing doxorubicin Transdrug(R) in HCC is based on the ability of the drug to bypass multi-drug resistance in vitro and to increase the efficacy in vivo by increasing tumour necrosis and tumour cell apoptosis. Doxorubicin is a potent and widely known anthracycline, registered for treatment of numerous cancers including HCC. As a result of its preferential hepatic distribution, proven efficacy in numerous susceptible or resistant tumour models, especially hepatic tumour models, Doxorubicin-Transdrug is being studied in a Phase I/II clinical trial utilizing the hepatic intra-arterial route of administration

About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC) is the fifth most common form of cancer, with an estimated 566,000 people worldwide (about 30,000 cases in Europe and 15,000 cases in the U.S.) diagnosed each year and an almost equal number of people dying from the disease. The 5-year survival rate of HCC is less than 5% without treatment, making it also one of the most deadly diseases. There are currently no approved therapies for the treatment of HCC. The only proven potentially curative therapy is surgical resection or liver transplantation.

The incidence of hepatocellular carcinoma is increasing worldwide, but striking geographical differences are observed in both risk factors and occurrence. The incidence in developing countries, particularly in East and Southeast Asia (especially in China and Japan) and Sub-Saharan Africa, is many times higher than in developed countries. Chronic infection with the hepatitis B virus (HBV) and hepatitis C virus (HCV) in the etiology of HCC is well established and has played a significant role in the increase of the disease. The increase in hepatitis incidence in Western countries explains the continuing increase of the disease in both Europe and the U.S. In Europe, 28% of HCC cases have been attributed to chronic HBV infection and 21% to HCV infection. Other risk factors such as alcohol consumption, cigarette smoking and oral contraceptives may explain the residual variations within countries.

Source: BioAlliance Pharma

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