ClolarTreatment for Acute Lymphoblastic Leukemia
FDA Advisory Committee Recommends Accelerated Clofarabine Approval for Most Common Pediatric Leukemia; FDA to Make Approval Decision by End of Year
WASHINGTON, December 1, 2004 - Bioenvision, Inc. (Nasdaq: BIVN) announced today that the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) positively endorsed clofarabine for the treatment of refractory or relapsed acute lymphoblastic leukemia (ALL). The panel advised conducting additional studies with clofarabine in acute myeloid leukemia (AML) before recommending an approval in this harder-to-treat leukemia, for which data was also submitted. The New Drug Application (NDA) for clofarabine, prepared and filed by Bioenvision's exclusive U.S. sublicensee, ILEX Products, Inc., a subsidiary of ILEX Oncology, Inc. (Nasdaq: ILXO), is under priority review by the FDA and has been granted fast track designation. Under the Prescription Drug User Fee Act, the FDA has until December 30, 2004 to complete the review of the clofarabine New Drug Application (NDA).
"This positive recommendation for the approval of clofarabine for the treatment of pediatric ALL is an endorsement for Bioenvision and Ilex's partnership in developing this new therapeutic agent for areas of unmet clinical need," said Dr Christopher Wood, CEO and Chairman of Bioenvision. "If approved by the FDA clofarabine will represent the first drug to be indicated initially for pediatric leukemia in more than a decade."
The committee based its opinion on data from pivotal Phase II acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) trials. The total ALL group (n=49) achieved a 31 percent overall response rate, with six complete remissions (CR), four complete marrow remissions in the absence of platelet recovery (CRp) and five partial remissions (PR). The total AML group (n=35) achieved a 26 percent response rate, with one CRp and eight PRs. Among both groups, 19 patients went on to receive bone marrow or stem cell transplants following their responses to clofarabine, which provides an opportunity to prolong life for these patients.
Bioenvision submitted a Marketing Authorization Application (MAA) with the European Medicines Evaluation Agency (EMEA) in July 2004 for the approval of clofarabine for the treatment of acute leukemia in children and young adults.
Clofarabine was previously granted orphan drug designation for the treatment of adult and pediatric ALL and AML by both the FDA and the EMEA. In the U.S., orphan drug status extends seven years of market exclusivity for the orphan drug indication following the FDA's marketing approval. The FDA also recently granted six months of extended market exclusivity to clofarabine under the Best Pharmaceuticals for Children Act. In Europe, the orphan drug designation provides European marketing exclusivity for 10 years.
Clofarabine is a next generation of the drug class purine nucleoside analogs which all inhibit DNA production necessary for cancer cell growth. Bioenvision, Inc. (NASDAQ:BIVN) sub-licensed ILEX the right to develop and market clofarabine for human cancer indications in the United States and Canada. Bioenvision is entitled to milestone payments tied to the development of the compound and is entitled to royalties on North American sales. As is its exclusive right, Bioenvision is developing clofarabine in the rest of the world. Bioenvision originally obtained clofarabine development and commercialization rights under patents held by Southern Research Institute.
Bioenvision's (Nasdaq: BIVN) primary focus is the development and distribution of compounds and technologies for the treatment of cancer. Bioenvision has a broad pipeline of products for the treatment of cancer, including: clofarabine (in co-development with ILEX Oncology, Inc.), Modrenal(R) (for which Bioenvision has obtained regulatory approval for marketing in the United Kingdom for the treatment of post-menopausal breast cancer following relapse to initial hormone therapy), and other products in clinical trials. Bioenvision is also developing anti-infective technologies, including the OLIGON technology; an advanced biomaterial that has been incorporated into various FDA approved medical devices.
Posted: December 2004
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