"As is often the case, studies such as these in rare patient populations provide insights into the disease and the patients' experience of their disease that are missing from the literature. While each patient's experience of his or her disease is personal and unique, there are common symptoms uncovered, sometimes under-recognized, that build and broaden the medical community's understanding and approach to treating the patient and his or her condition," said Dr.
As part of its efforts to better capture the burden of illness experienced by FCS patients, Akcea recently launched the IN-FOCUS study at www.fcsinfocus.com. Information gathered from IN-FOCUS can help raise awareness about this rare, severe disease, lead to a better understanding of the burden of this disease and inform treatment approaches for patients with FCS.
Akcea and its collaborators will present the following two posters at the ASPC meeting.
One poster titled "Characterizing Familial Chylomicronemia Syndrome: Baseline Data of the APPROACH Study" presents data from patients participating in APPROACH, the pivotal Phase 3 study evaluating the efficacy and safety of volanesorsen for the treatment of patients with FCS. The findings from this poster provide insights on the diversity of the overall patient population and the clinical characteristics of the disease.
A second poster titled "The Clinical and Psychosocial Burden of Patients with Familial Chylomicronemia Syndrome" presents findings from a facilitator-moderated session, which details the impact of FCS on multiple quality of life measures for patients and caregivers.
ABOUT FAMILIAL CHYLOMICRONEMIA SYNDROME (FCS)
FCS is a rare, genetic disorder and may also be called familial chylomicronemia or Fredrickson Type 1 hyperlipoproteinemia, or familial lipoprotein lipase deficiency. People with FCS are unable to effectively clear lipid particles called chylomicrons. As a result, they have extremely high levels of triglycerides and are at risk of significant morbidity and mortality, including potentially life-threatening pancreatitis. To learn more about FCS visit fcsfocus.com and to learn about the
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics is focused on developing and commercializing drugs for patients with serious cardiometabolic diseases caused by lipid disorders. Established as a wholly owned subsidiary of
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over a dozen drugs in mid- to late-stage development. Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
This press release includes forward-looking statements regarding the business of
In this press release, unless the context requires otherwise, "Akcea," "Company," "we," "our," and "us" refers to Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of
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D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741