An Open Letter to Familial Chylomicronemia Syndrome Patients Regarding Access to Therapy

Last Updated: Tuesday, 18-Dec-2018 19:00:00 EST


December 18, 2018
Melissa Goetz
Lindsey Sutton
Familial Chylomicronemia Syndrome Foundation
7523 Rowena St
San Diego, CA 92119
An Open Letter to Familial Chylomicronemia Syndrome Patients Regarding Access to Therapy
As you know, the National Lipid Association (NLA) is a nonprofit, multidisciplinary medical society focused on enhancing the practice of lipid management in clinical medicine, representing nearly 2,250 members throughout the United States. The NLA is the leader in this field, having published several patient-centered recommendations for lipid management and serving as the primary educator and advocate for lipidology. The Foundation of the NLA is dedicated to improving the welfare of patients and families affected by cholesterol and triglyceride problems by supporting patients, families, and providers through education, awareness, and advocacy in order to decrease suffering and death.
As clinicians with the privilege of taking care of patients with Familial Chylomicronemia Syndrome (FCS), we support your efforts to urge the United States Food and Drug Administration (FDA) to approve volanesorsen, particularly in light of there currently being no other effective medical therapy for FCS.
The NLA and Foundation of the NLA recognize that FCS patients are severely ill, and that despite aggressive diet, have significant morbidity from their disease.  By bringing patients with FCS together to discuss their disease, the major effect on quality of life has become more apparent. These quality of life issues have not been well appreciated in the past.  Although some patients respond well to dietary therapy, we realize that the diet required has a significant negative impact on a patient’s (and his or her family’s) lifestyle and is often insufficient to manage the patient’s wellbeing. For those patients who do not respond well to dietary therapy, there is little to offer currently to reduce recurrent hospitalization for abdominal pain and pancreatitis.
We recognize that there are several safety concerns with volanesorsen, including severe thrombocytopenia or injection site reactions, but it seems these can be effectively managed by experts in clinical lipidology and the FCS patients, both of whom are highly motivated due to the severe symptoms experienced by these patients.
We support your efforts to call upon the FDA and Akcea Therapeutics to find an acceptable regulatory path forward that can expedite the approval of volanesorsen, which is desperately needed for this patient population with a significant unmet medical need. Please reach out to the NLA and Foundation of the NLA for support whenever you feel it necessary in your journey towards the availability of an effective therapy.
Respectfully submitted on behalf of the NLA and Foundation of the NLA,
Alan S. Brown, MD, FNLA
National Lipid Association
Anne C. Goldberg, MD, FNLA
Foundation of the National Lipid Association

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