Pharmion Corporation (Nasdaq: PHRM) announced today the enrollment of the first patient in AVIDA(TM), a registry for patients treated with Vidaza(R) (azacitidine for injectable suspension). Vidaza was approved in May 2004 for the treatment of all five French-American-British (FAB) subtypes of myelodysplastic syndromes (MDS): refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) if accompanied by neutropenia or thrombocytopenia or requiring transfusions; refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL). Myelodysplastic syndromes are a serious and life-threatening group of diseases in which the bone marrow does not function normally, resulting in the production of malformed or immature blood cells.
The AVIDA patient registry is a prospective, multi-center, observational study compiling data on the natural history of MDS and management of patients receiving Vidaza. The registry is designed to provide insights into the diagnosis and management of MDS and its treatment and outcomes in a real-world setting. Additionally, patients will be able to provide feedback on their treatment satisfaction and its impact on their quality of life.
The primary objectives for the registry are to describe current usage patterns for Vidaza in the community, including the use of concomitant care procedures and treatment used in conjunction with Vidaza, and to correlate duration of therapy and the number of treatment cycles with clinical response. Data generated from this study will serve as a foundation to support further clinical development initiatives, and a robust publications plan will result in the dissemination of information related to treatment best practices.
“Results from the AVIDA registry will be of direct interest to hematology/oncology physicians across the country,” said Dr. David Grinblatt, Scientific Advisory Board Chairperson for AVIDA and Associate Professor of Medicine at the Feinberg School of Medicine of Evanston Northwestern Healthcare. “It will help describe the effect on bone marrow function and document the impact on patient satisfaction and quality of life from the current use of Vidaza in the community.”
Physician hematologist/oncologists in approximately 100 to 150 community-based oncology centers will participate and enroll a total of 300 to 500 patients. The patients will be followed for up to two years each. The Company plans to publish data from the registry on an ongoing basis. The AVIDA registry study is supported by a Scientific Advisory Board (SAB) that will provide guidance on clinical issues including data interpretation and analytics reviews. In addition, the registry study is HIPAA-compliant and a central Institutional Review Board has been selected to provide study oversight.
“We are very enthusiastic about the launch of this registry as part of our ongoing and significant commitment to the post-market evaluation of Vidaza,” said Patrick Mahaffy, president and chief executive officer of Pharmion. “We believe the resulting data will provide practical information to physicians in the community, providing a greater understanding of how Vidaza is used to provide clinical benefit to their patients.”
On May 19, 2004, Vidaza (azacitidine for injectable suspension) became the first drug approved by the FDA for the treatment of patients with Myelodysplastic Syndromes (MDS). The FDA approved Vidaza, the first in a new class of drugs called demethylation agents, for treatment of all five FAB MDS subtypes, which include both low-risk and high-risk patients. These subtypes include: refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) if accompanied by neutropenia or thrombocytopenia or requiring transfusions; refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL).
Vidaza is believed to exert its antineoplastic effects by causing hypomethylation of DNA and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. The concentration of azacitidine required for maximum inhibition of DNA methylation in vitro does not cause major suppression of DNA synthesis. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The cytotoxic effects of azacitidine cause the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. Non-proliferating cells are relatively insensitive to Vidaza.
Important Safety Information
Vidaza is contraindicated in patients with a known hypersensitivity to azacitidine or mannitol and in patients with advanced malignant hepatic tumors.
In clinical studies, the most commonly occurring adverse reactions were nausea (70.5%), anemia (69.5%), thrombocytopenia (65.5%), vomiting (54.1%), pyrexia (51.8%), leukopenia (48.2%), diarrhea (36.4%), fatigue (35.9%), injection site erythema (35.0%), constipation (33.6%), neutropenia (32.3%) and ecchymosis (30.5%). Other adverse reactions included dizziness (18.6%), chest pain (16.4%), febrile neutropenia (16.4%), myalgia (15.9%), injection site reaction (13.6%), aggravated fatigue (12.7%) and malaise (10.9%).
Because treatment with Vidaza is associated with neutropenia and thrombocytopenia, complete blood counts should be performed as needed to monitor response and toxicity, but at a minimum, prior to each dosing cycle.
Because azacitidine is potentially hepatotoxic in patients with severe pre-existing hepatic impairment, caution is needed in patients with liver disease. In addition, azacitidine and its metabolites are substantially excreted by the kidneys and the risk of toxic reactions to this drug may be greater in patients with impaired renal function. Because elderly patients are more likely to have decreased renal function, it may be useful to monitor renal function.
Vidaza may cause fetal harm. While receiving treatment with Vidaza, women of childbearing potential should avoid becoming pregnant, and men should avoid fathering a child. In addition, women treated with Vidaza should not nurse.
Pharmion is a biotechnology company focused on acquiring, developing and commercializing innovative products for the treatment of hematology and oncology patients in the U.S., Europe and additional international markets. Pharmion has a number of products on the market including the world’s first approved epigenetic drug, Vidaza(R), a DNA demethylating agent.