Sponsor Overview
Explore verified public information about Ionis Pharmaceuticals, Inc.'s expanded access programs, compassionate use policies, and patient eligibility pathways. This report aggregates official statements, independent research, and regulatory data to help patients, providers, and sponsors collaborate efficiently.
Unique references curated across 3 content categories.
ClinicalTrials.gov data sources power this status.
Dataset refreshed continuously through Right2Hope ETL pipelines.
Expanded Access Intelligence
Official Statements
Score contribution: 100 — 1 supporting sources.
“Currently, Ionis does not have any investigational products available for Expanded Access.”
Active EAPs on ClinicalTrials.gov
Score contribution: 60 — 1 supporting sources.
Conditions: Hereditary Angioedema
Past EAPs on ClinicalTrials.gov
Score contribution: 40 — 2 supporting sources.
Conditions: Familial Chylomicronemia Syndrome
Conditions: Amyloidosis, Hereditary
Reagan-Udall Foundation Insights
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.
Partner with Right2Hope
Right2Hope helps sponsors translate regulatory programs into actionable patient access pathways. Connect with our team to publish new programs, streamline intake workflows, or align with providers seeking compassionate use options.