The purpose of the Expanded Access Program is to provide pre-approval access of olezarsento eligible patients with Familial Chylomicronemia Syndrome (FCS).
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/
Drug: Olezarsen
Olezarsen 80 mg administered once monthly by subcutaneous (SC) injections in the abdomen,
thigh, or upper arm.
Other Name: ISIS-678354
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.
Expanded Access Site
Carlsbad, California, United States
Not Provided