Ionis Pharmaceuticals, Inc.

Expanded Access Intelligence

Official Statements

Score contribution: 100 1 supporting sources.

Score 1001 references
Featured Reference

Currently, Ionis does not have any investigational products available for Expanded Access.

Active EAPs on ClinicalTrials.gov

Score contribution: 60 1 supporting sources.

Score 601 references
Featured Reference

Conditions: Hereditary Angioedema

Past EAPs on ClinicalTrials.gov

Score contribution: 40 2 supporting sources.

Score 402 references
Featured Reference

Conditions: Familial Chylomicronemia Syndrome

Conditions: Amyloidosis, Hereditary

Reagan-Udall Foundation Insights

Company
Ionis Pharmaceuticals, Inc.
Additional Information

Available Therapies via Single-Patient EA Olezarsen for Familial Chylomicronemia Syndrome (FCS) At present, Ionis Pharmaceuticals has an EAP available for olezarsen (ClinicalTrials.gov ID NCT06360237). Olezarsen is an investigational antisense oligonucleotide-GalNAc3 conjugate that causes degradation of apoC-III mRNA and a reduction of serum apoC-III protein. Studies suggest that apoC-III regulates both triglyceride metabolism and hepatic clearance of chylomicrons and other triglyceride-rich lipoproteins. The expanded access program for olezarsen is intended for those at risk of severely elevated triglyceride levels, particularly those living with FCS. The program is currently only available in the United States. If you are a patient, family member or caregiver and you wish to know more about the olezarsen EAP for FCS, please discuss the EAP and all treatment options with your treating physician. If you are a treating physician and are seeking information about the olezarsen EAP or would like to request access for a patient, please contact patient.access@bionicalemas.com . Bionical Emas is a Clinical Research Organization managing the olezarsen EAP on behalf of Ionis. Safety and efficacy have not been evaluated or established by any regulatory authorities for olezarsen.

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