Press Releases
Akcea Therapeutics Announces Launch of IN-FOCUS, a Research Study to Assess Impact of Familial Chylomicronemia Syndrome
FCS, also known as lipoprotein lipase deficiency (LPLD) or type 1 hyperlipoproteinemia, is a rare, genetic disease characterized by extremely high levels of triglycerides, the risk of pancreatitis and multiple other complications, which impact patients' daily lives. To date, a holistic assessment of this impact has never been measured from the patient's perspective.
"By participating in the IN-FOCUS study, people living with FCS have a historic opportunity to help other patients, as well as clinicians and care providers, better understand this rare but very serious condition," said Dr.
Participants will be asked a series of questions to confirm that only patients who are living with FCS are captured in the study. Adults over 18 years of age living with FCS who qualify will then be invited to complete an anonymous survey by navigating directly to www.fcsinfocus.com. Participants will be asked to:
- share information about how FCS affects different aspects of their life;
- document experiences related to their symptoms, co-morbidities, health complications, hospital visits, etc.; and,
- assess whether and how well FCS can be managed by modifications in diet and lifestyle alone.
The survey is anonymous; at no time will participants be asked to reveal any self-identifying information. They will also have the option to respond "Don't Know" to queries if they are uncomfortable sharing any of the requested information.
All people 18 years and older living with FCS are encouraged to participate. Clinicians and care providers working with the FCS community are invited to share information about the survey with patients and encourage them to consider participating.
"This research will help advance medical understanding of diseases of lipid metabolism, especially those that have been under-recognized or for which there are critical gaps in the medical literature," according to Dr.
"Many patients and clinicians have confirmed details about the life-altering experience of having FCS, including periods of persistent severe pain, fear of developing pancreatitis, extreme fatigue and many psychosocial challenges, but precise and insightful assessments of disease burden from the patients' perspective have not been captured," said Dr. O'Dea. "As a company focused on transforming the lives of patients with serious cardiometabolic diseases caused by lipid disorders, we at Akcea are committed to advancing understanding of these diseases, both through our clinical trials, as well as through important medical initiatives such as the IN-FOCUS study."
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. Additional information on FCS is available at www.fcsfocus.com.
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
ABOUT
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.
FORWARD-LOOKING STATEMENT
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.
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SOURCE
Investor and Media:D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741