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FDA Grants Orphan Drug Designation for Doxorubicin Transdrug, Treatment for Primary Liver Cancer

April 25, 2005 -- PARIS--(BUSINESS WIRE)-- BioAlliance Pharma, a biopharmaceutical company focused on the field of drug resistance, announced today that one of its lead products, doxorubicin Transdrug(R), has been granted orphan drug status for the treatment of a deadly form of liver cancer, hepatocellular carcinoma, by the U.S. Food and Drug Administration (FDA).

The FDA decision is important to patients suffering from HCC and to BioAlliance Pharma because orphan drug designation creates favorable conditions for the development of drugs for diseases affecting less than 200,000 people in the United States, including financial incentives for the company involved. A similar designation, with similar advantages, was granted in the European Union by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA) in late 2004.

BioAlliance's doxorubicin Transdrug(R) is currently completing Phase I/II clinical testing in the EU for treatment of hepatocellular carcinoma (HCC), a form of liver cancer for which there is no approved therapy. The company's Transdrug technology employs a proprietary polymer formulated as a nanoparticle to deliver a proven active cancer drug (doxorubicin) to the site of the liver tumor via a catheter placed in the hepatic artery.

"The orphan drug designation for doxorubicin Transdrug(R) in the U.S. is another important milestone for patients suffering from HCC and for BioAlliance," said Dominique Costantini, M.D., president and CEO of BioAlliance Pharma. "There are currently no approved therapies for the treatment of hepatocellular carcinoma, and the only proven potentially curative options are surgical resection or liver transplantation. Orphan drug designation in both the U.S. and the EU makes development commercially viable and provides multiple incentives for BioAlliance to undertake further clinical trials with the goal of developing an effective therapy for this deadly disease."

About Doxorubicin Transdrug
BioAlliance has developed a proprietary technology using PIHCA (poly-iso-hexyl-cyanoacrylate), a proprietary polymer to formulate a number of anti-cancer drugs in nanoparticulate form. In the human body, these drug-loaded nanoparticles, which can be formulated for delivery of drugs through the intra-arterial, intravenous, or oral route of administration, are translocated into the cancer cell where they can elicit their known anti-cancer activity. Hence the name of the technology: Transdrug(R).

Resistance has become a serious problem in cancer treatment, and multi-drug resistance (MDR) at present is the main cause of chemotherapy treatment failure. Transdrug(R), which is designed to bypass MDR mechanisms, is capable of restoring the sensitivity of cancer cells, overcoming resistance in cancer therapy, and may thereby fill a very important therapeutic gap in cancer treatment (X Pepin 1997).

In particular, the tumors associated with hepatocellular carcinoma can become resistant to chemotherapeutic agents (E Pang 2005), which results in a loss of response to further therapy. This resistance is likely the result of multidrug resistance P-glycoproteins and MDR-associated proteins together with reduced hepatocellular drug uptake into the tumors (G Zollner 2005).

BioAlliance is completing a Phase I/II clinical trial with doxorubicin Transdrug(R) in HCC at eight major clinical sites in France. Since HCC is a resistant cancer with a poor prognosis, it represents an important indication for doxorubicin Transdrug(R) which is under investigation to demonstrate preferential distribution in the liver and efficacy in circumventing cancer resistance.

Cytotoxic agents, such as doxorubicin, have been used extensively to treat cancer. However, the non-specificity of these drugs toward healthy cells in addition to cancer cells has curtailed their administration at the most therapeutic or efficacious levels. Therefore, developing a delivery system that specifically targets tumor cells, reducing toxicity to healthy cells and overcoming drug resistance, would constitute a significant advance in treating various cancers with standard chemotherapy.

About Orphan Drug Designation
In the U.S., orphan drug designation provides a drug U.S. market exclusivity for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. Other incentives include protocol assistance and eligibility for research and development support. Protocol assistance includes regulatory assistance as well as advice on the conduct of clinical trials.

The designation does not shorten the duration of the regulatory review and approval process. If a product that has orphan drug designation is the first such product to receive FDA approval for the disease/indication for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug in the U.S. for the same disease, except in limited circumstances, for seven years. The FDA may permit additional companies to market the same drug for the designated condition if such companies can demonstrate better safety, efficacy, or a major contribution to patient care. The FDA may also approve more than one product for the same orphan indication or disease as long as the products are different drugs.

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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