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FDA Advisory Committee Votes Unanimously To Recommend Approval Of Novartis Corporation's (NVS) Exjade(R)



10/19/2005 5:13:08 PM

EAST HANOVER, N.J., Sept. 29 /PRNewswire-FirstCall/ -- Novartis announced today that the Blood Products Advisory Committee (BPAC) of the U.S. Food and Drug Administration (FDA) gave a positive review of Exjade(R) (deferasirox).

Exjade is currently under priority review by the FDA as the first and only once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions in adults and children. A newly designed molecule, Exjade is administered as a once-daily drink after tablet(s) are dispersed in a glass of water or orange juice.

The committee unanimously voted to recommend approval for use of Exjade for patients with chronic iron overload due to blood transfusions. The FDA generally follows the recommendations of its advisory committees, although it is not obligated to do so. If approved, Exjade may be a major advance in iron chelation therapy in the U.S.

"We believe the Blood Products Advisory Committee has recognized the potential of Exjade to dramatically improve the management of patients with transfusional iron overload," said Diane Young, MD, vice president, global head, Clinical Development, Novartis Oncology. "During the next several weeks we will work closely with the FDA to answer any remaining questions, in hopes of bringing this new treatment option to patients as soon as possible."

Exjade, which has been designated an orphan drug in both the EU and the US, is currently under priority review in the US, Canada, Switzerland, Australia and New Zealand. Additional regulatory submissions have been filed around the world.

Iron overload is a cumulative, potentially life-threatening, unavoidable consequence of frequent blood transfusions used to treat certain rare, chronic blood disorders, including thalassemia, sickle cell disease, other rare anemias and myelodysplastic syndromes. Signs of iron overload may be detected after 10 to 20 blood transfusions. If left undiagnosed or untreated, the excess iron in the body leads to damage to the liver, heart and endocrine glands. Iron chelation has been demonstrated to be the only effective treatment for transfusion-related iron overload.

Filing Data

The Exjade filings were based on the results of a pivotal clinical trials program, including a Phase III head-to-head trial vs. Desferal(R) (deferoxamine), which showed that Exjade significantly reduced liver iron concentration (LIC) at doses of 20-30 mg/kg/day. These clinical trials, which included more than 1,000 adults and children, were part of the largest prospective global clinical trials program ever implemented for an investigational iron chelator. LIC is the accepted indicator for total body iron content in patients receiving blood transfusions. The studies demonstrated that Exjade at 20-30 mg/kg/day led to the maintenance or reduction of iron burden in transfused patients with thalassemia, sickle cell disease, other rare anemias and myelodysplatic syndromes. In the clinical studies, Exjade was generally well tolerated, with the most frequently reported adverse events being nausea, vomiting, diarrhea, abdominal pain, skin rash and increases in serum creatinine. As with deferoxamine, cases of ocular and auditory disturbances have been reported.

About Iron Chelation

In iron chelation an agent binds to iron in the body and helps remove it through the urine and/or feces. To date, only deferoxamine is globally available for the first-line treatment of transfusional iron overload. While deferoxamine is effective, it 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 or has excess iron within their body. In many patients the need for transfusions may be life-long. However, due to the inconvenience and discomfort associated with the administration of deferoxamine, many patients choose not to undergo iron chelation therapy, exposing themselves to the dangers of iron overload. Novartis believes the approval of Exjade would, therefore, not only help patients currently receiving iron chelation, but also extend the benefits of iron chelation to those not currently undergoing therapy.

Additional Information

Exjade is still in clinical development and not yet approved by the U.S. FDA. The brand name "Exjade" is also subject to approval by the FDA. Some patients may be eligible to enroll in ongoing clinical trials. To learn more about Exjade clinical trials, patients, caregivers and their health care providers can call 1-800-340-6843, Monday through Friday, between 8:30 am and 5:30 pm Eastern Time.

The foregoing release contains forward-looking statements that can be identified by terminology such as "if approved, Exjade may be," "believes," "potential," "significantly impact," "will," "in hopes of bringing," "would," "dramatically improve," "recommended," or similar expressions, or by express or implied discussions regarding potential 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 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 U.S. 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. 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.

Full prescribing information on Desferal (deferoxamine) is available at http://www.pharma.us.novartis.com/product/pi/pdf/desferal.pdf.

About Novartis

Novartis Pharmaceuticals Corporation researches, develops, manufactures and markets leading innovative prescription drugs used to treat a number of diseases and conditions, including central nervous system disorders, organ transplantation, cardiovascular diseases, dermatological diseases, respiratory disorders, cancer and arthritis. The company's mission is to improve people's lives by pioneering novel healthcare solutions.

Located in East Hanover, New Jersey, Novartis Pharmaceuticals Corporation is an affiliate of Novartis AG -- a world leader in pharmaceuticals and consumer health. In 2004, the Group's businesses achieved sales of USD 28.2 billion and pro forma net income of USD 5.6 billion. The Group invested approximately USD 4.2 billion in R&D. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 83,700 people and operate in over 140 countries around the world. For further information please consult http://www.novartis.com.

Contacts: Kim Fox Novartis Oncology Tel +1 862 778 7692 Mobile +1 973 960 7532 kim.fox@novartis.com

Novartis Pharmaceuticals Corporation

CONTACT: Kim Fox of Novartis Oncology, +1-862-778-7692, mobile:+1-973-960-7532, or kim.fox@novartis.com, for Novartis PharmaceuticalsCorporation


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