Sponsor Overview
Explore verified public information about Ultragenyx Pharmaceutical 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 — 3 supporting sources.
“We recognize that some patients with serious or immediately life-threatening diseases may not be able or eligible to participate in a clinical trial and may not have other treatment options available to them. Our early access program makes our investigational therapies available to qualified patients with critical and compelling need.”
“In those cases, we make our investigational therapies available to qualified patients under certain circumstances of critical and compelling need through our early access program.”
“We also provide early pre-approval access to our therapies when medically appropriate.”
Active EAPs on ClinicalTrials.gov
Score contribution: 60 — 2 supporting sources.
Conditions: MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
Conditions: Long Chain Fatty Acid Oxidation Disorders
Reagan-Udall Foundation Insights
Single-Patient EA Policies/Criteria Requests for early access to investigational therapies must be made by a qualified physician.
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