Press Releases
Isis Announces Positive Decision on European Orphan Drug Designation for ISIS-APOCIII Rx
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"FCS is a rare and very serious genetic disorder that is often associated with triglyceride levels higher than 2,000 mg/dL. Orphan drug designation for FCS underscores the need for improved therapies to treat patients with FCS. Because of their extremely high levels of triglycerides, FCS patients are at significant risk of many serious health conditions, including frequent episodes of pancreatitis, which can require hospitalization and be life-threatening. Current treatment options do not reduce triglyceride levels enough to reduce the risk of serious illness in patients with FCS," said
The COMP, a committee of the
ISIS-APOCIIIRx is an antisense drug intended to treat patients with severely high triglycerides either as a single agent or in combination with other triglyceride-lowering agents. ISIS-APOCIIIRx targets apoC-III, a protein produced in the liver that plays a central role in the regulation of serum triglycerides. Isis is developing ISIS-APOCIIIRx to treat patients with FCS and patients with severely elevated triglycerides. FCS is a rare genetic disorder associated with extremely high triglyceride levels, often triglyceride levels that are higher than 2,000 mg/dL, that affects an estimated 3,000 to 5,000 patients worldwide. As a result of their severely high triglyceride levels, FCS patients are at risk of many serious health conditions, including recurrent acute pancreatitis that often requires hospitalization, enlargement of the liver and spleen, abdominal pain and eruptive fatty skin lesions. Isis is also developing ISIS-APOCIIIRx for patients with severely elevated triglycerides, greater than 880 md/dL, that affects an estimated 50,000 patients in
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Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 31 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer. Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO®, in
This press release includes forward-looking statements regarding the discovery, development, activity, therapeutic potential and safety of ISIS-APOCIIIRx. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended
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SOURCE
D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741, or Amy Blackley, Ph.D., Associate Director, Corporate Communications, 760-603-2772