- Innovative, once-a-day oral formulation addresses unmet medical need for a broad range of iron overload patients
Dorval, Quebec, July 14, 2005 – Novartis Pharmaceuticals Canada Inc. announced today that Health Canada has granted a Priority Review for Exjade™ (deferasirox), a once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions. In Canada, a Priority Review is generally reserved for drugs intended for the treatment of a serious or life-threatening condition that demonstrate the potential to address unmet medical needs for that condition. Based on clinical trial data, Exjade™ has the potential to provide a significant clinical advantage over existing therapy.
Exjade™ is a dispersible tablet that is mixed in water and taken once daily as iron chelation therapy, which is the removal of excess iron. The current standard of care in iron chelation is Desferal® (deferoxamine). Desferal® is effective when taken as prescribed, but typically requires subcutaneous infusion lasting eight to twelve hours per day, for five to seven days a week for as long as the patient continues to receive blood transfusions. In many patients, the need for transfusion and chelation therapy may be life-long.
"Patients are grateful that Health Canada recognized the urgency of a once-daily oral chelator,” said Durhane Wong-Rieger, PhD, president and chief executive officer, Anemia Institute for Research and Education in Canada. "Older teens and young adults in particular will often not comply with Desferal® therapy because it is so burdensome. This increases the risks of iron overload which include deformities, organ failure and even premature death. Obviously, we hope that the Priority Review means that Exjade™ will soon be available to all patients who are regularly transfused and require iron chelation therapy."
A priority review means that a drug could be approved within 200 days, as opposed to the average two years, making it available that much sooner to patients. Exjade™, also known as investigational agent ICL670, has also been granted priority review in Australia and the U.S., as well as fast track status in Switzerland. Furthermore, Exjade™ has received Orphan Drug** status in the U.S., EU and Australia.
Iron overload causes cumulative life-threatening toxicity that results from lifesaving blood transfusions required to treat certain types of anemias and other disorders, including thalassemia, sickle cell disease, other rare anemias, and myelodysplastic syndromes. If left undiagnosed or untreated, iron overload can damage the liver, heart and endocrine glands.
Filing data
The Exjade™ global clinical trials program enrolled more than 1,000 patients, and is the largest-ever prospectively implemented for an investigational iron chelator. The filings are based on the results of pivotal clinical trials, including a Phase III head-to-head trial vs. deferoxamine, which showed that at doses of 20 mg and 30 mg/kg Exjade™ significantly reduced liver iron concentration (LIC) in adult and pediatric patients receiving blood transfusions. Findings from the clinical trial program were presented in December 2004 at the annual meeting of the American Society of Hematology. The studies demonstrated that Exjade™ led to the maintenance or reduction of absolute LIC in regularly transfused patients with different underlying diseases.
In the clinical studies in both adults and children as young as two years of age, Exjade™ was generally well tolerated, with the most frequently reported adverse events being nausea, vomiting, diarrhea, abdominal pain, skin rash, a mild to moderate increase in liver enzymes and mild stable increases in serum creatinine, usually within the normal range. Health Canada is currently reviewing the safety and efficacy data as part of the Priority Review.
**Orphan drug designation and fast-track status
In the EU, the filing for Exjade™ was submitted to the European Medicines Agency under the centralized procedure. Exjade™ was granted Orphan Drug status in both the US and EU in 2002. The intent of the Orphan Drug designation is to stimulate the research, development, and approval of products that treat rare diseases. In the EU, the term “Orphan Drug” refers to a product that treats a serious or life-threatening disease that affects fewer than five people per 10,000 population. In the US, the term “Orphan Drug” refers to a product that treats a disease that affects fewer than 200,000 people in the country. Exjade™ also was granted fast-track status in the US and Switzerland. In Canada, an Orphan Drug policy does not exist. However, a Priority Review status has been granted for Exjade™. A Priority Review designation is generally reserved for drugs intended for the treatment of a serious or life-threatening condition that demonstrate the potential to address unmet medical needs for that condition.
Forward looking statement
The foregoing release contains forward-looking statements that can be identified by terminology such as “innovative,” “convenient,” or similar expressions, or by express or implied discussions regarding potential additional marketing approvals or future sales of Exjade™. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results with Exjade™ to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Exjade™ will receive any additional marketing approvals in any other countries, or that it will reach any particular sales levels. In particular, management's expectations regarding commercialization of Exjade™ could be affected by, among other things, additional analysis of Exjade™ clinical data, new clinical data, unexpected clinical trial results, unexpected regulatory actions or delays or government regulation generally, the company's ability to obtain or maintain patent or other proprietary intellectual property protection, competition in general, increased government, industry, and general public pricing pressures, and other risks and factors referred to in the Company's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. The purpose of this media document is for information only, and is not meant to promote or encourage a use of this medication outside the approved Product Monograph in Canada. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
