Familial Chylomicronemia Syndrome
Conditions
Keywords
Familial Chylomicronemia, Familial Lipoprotein Lipase Deficiency, Hyperlipoproteinemias, Familial Hyperlipoproteinemia Type 1, Hyperlipoproteinemia Type 1, Hyperchylomicronemia, Familial, Lipoprotein Lipase Deficiency, Familial, Hyperlipidemias, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Metabolic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn
Brief summary
The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).
Detailed description
The Expanded Access Program (EAP) is intended to provide pre-approval access to olezarsen for eligible patients with FCS who have limited or no available treatment options. This program is open in the United States and operates under the individual patient (also referred to as single patient) IND expanded access route in which the patient's treating physician serves as the sponsor. Expanded access requests must be from the patient's treating physicians and submitted according to the instructions at https://www.ionispharma.com/patients/expanded-access-policy/
Interventions
DRUGOlezarsen
Olezarsen 80 mg administered once monthly by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.
Sponsors
Ionis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Inclusion criteria
* Has a diagnosis of FCS as determined by the sponsoring physician. Ionis will review each application to determine eligibility based on documentation of validated genetic or clinical diagnosis. o Documented loss of function mutations (homozygous, compound / double heterozygous) in genes such as LPL, GPIHBP1, APOA5, APOC2 or LMF1) or clinically validated diagnosis of FCS. * Resides in and is a resident of the United States. * Willing to follow a diet comprising ≤20 g fat per day.
Exclusion criteria
* Has any new or worsening of existing conditions which, in the opinion of the physician, would make the patient unsuitable for treatment with olezarsen. * Olezarsen naïve patients with baseline platelet count \<100x109/L at qualification. * Estimated GFR (eGFR) \<30 mL/min/1.73 m2. * Secondary factors are the cause of triglyceride elevations. * Is currently hospitalized in an acute emergency setting.
Countries
United States
Outcome results
None listed