Pharmsynthez today announced that the United States Food and Drug Administration (FDA) has unanimously awarded Virexxa, OJSC Pharmsynthez’s treatment for hormonotherapy-resistant endometrial cancer (a malignant tumor of the female reproductive system), orphan drug designation.
Virexxa is the first Russian pharmaceutical to be awarded the status of orphan drug in the United States. The market capacity for this product in the US is estimated at US$ 500 million per year. The FDA made its decision based on a positive ruling by the Committee for Orphan Medicinal Products that combined therapy based on Virexxa can provide considerable clinical advantage to patients with endometrial cancer that is resistant to hormonotherapy.
According to David Frank MD, PhD , senior analyst for Leerink Swann investment bank (Boston, USA), “Virexxa is the only medicine in clinical trials for treatment of advanced and relapsing forms of endometrial cancer that works by increasing endometrial cancer’s sensitivity to hormonotherapy by means of induction of progesterone receptors in progesterone-tolerant endometrial cancer. The FDA‘s decision confirms Virexxa’s status as a unique niche product and significantly strengthens potential for commercialization of the medicine in the United States.”
Pharmsynthez is currently investigating the effectiveness of the combined application of Virexxa and progestins in patients with advanced and relapsing endometrial cancer who developed a tolerance to progestins monotherapy. Phase II clinical trials are ongoing in several clinical centers in the Russian Federation. Phase II clinical trials in the European Union countries will be initiated in 2011 after the completion of manufacturing of the cGMP medicine series for clinical trials by KeveltAS, an enterprise acquired by Pharmsynthez earlier in 2011. As a result of the FDA’s decision to grant orphan drug designation, Pharmsynthez will also initiate clinical trials of Virexxa in the US in 2011, one year earlier than previously planned.
Dmitry Genkin, the Chairman of the Board of Directors of Pharmsynthez, said: “The assignment of orphan drug status in the USA for Virexxa is a highly important event for Pharmsynthez. It serves as recognition that our unique product has the potential to address a serious unmet medical need and is competitive in the high value US pharmaceutical market. In addition to guaranteeing our exclusive market position for seven years following registration, orphan drug designation reduces the time and expenses required to complete the clinical trial program for Virexxa in the US and so allows us to begin clinical trials in the US this year, earlier than originally planned.”
In the USA orphan drug status is granted to a medicinal product intended to cure diseases which are dangerous to life or chronically disabling with a prevalence of less than 50,000 cases per year. It is assumed that there are no alternative methods of treatment of such diseases in the USA or the proposed drug should have considerable advantages over existing methods of treatment.
Orphan drug status gives the manufacturer substantial advantages for development, registration and marketing of the drug in the USA:
* The help of FDA experts in the development and examination of clinical trial reports, including free federal financing and tax credits for carrying out of clinical trials in the USA covering up to 50% of expenses.
* Simplified drug registration in the USA, making marketing of the drug possible after phase II clinical trials and a waiver of registration fees.
* Exclusive rights to drug manufacture and marketing for 7 years after the drug’s introduction to the US market. At the same time, the drug is fully covered by insurance programs.
About Receptor-Negative Endometrial Cancer, its Treatment and Unsatisfied Needs of Medicine
More than 20,000 women die annually from advanced and relapsing forms of endometrial cancer in the Western Europe and the USA. Chemotherapy and progestin hormone therapy constitute the only available treatment for advanced and relapsing forms of endometrial cancer. However, in both cases the survival rate is low after the initiation of treatment: less than one year. This is due to the development of resistance to chemotherapy and hormonal treatments. Hormonotherapy remains the preferred treatment method due to the considerably lower toxicity compared with chemotherapy. Previous clinical trials have shown that Virexxa helps to restore sensitivity of endometrial cancer to hormonal treatments from the progestin group, which opens new opportunities for hormonotherapy of advanced and relapsing forms of endometrial cancer.