Sponsor Overview
Explore verified public information about Esperare'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.
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Expanded Access Intelligence
Official Statements
Score contribution: 100 — 2 supporting sources.
“**Requests for compassionate use/expanded access will be considered on a case-by-case basis**, in accordance with applicable regulations and in alignment with our mission and values and with the safety of the patient and childbearing mother as a priority. Of note, ER-004 is delivered to the patient before birth by injection into the amniotic fluid, an untried route of administration that requires careful consideration in each individual case.”
“Les demandes d’Usage Compassionel/Expanded Access seront examinées au cas par cas, conformément aux réglementations applicables et à notre mission et à nos valeurs et en accordant la priorité à la sécurité du patient et de la mère. Il est à noter que le traitement ER-004 est administré au patient avant la naissance par injection dans le liquide amniotique, une voie d'administration non éprouvée qui nécessite d’examiner attentivement chaque cas individuellement.”
Reagan-Udall Foundation Insights
Single-Patient EA Policies/Criteria ER-004 COMPASSIONATE USE/EXPANDED ACCESS POLICY in XLHED Expanded access/Compassionate use refers to the use of an investigational therapy, that have not yet been approved by government regulatory agencies, outside a clinical trial, for patients with serious or life- threatening diseases or conditions who lack therapeutic alternatives. Esperare’s mission is to advance treatments for rare diseases and to promote treatment accessibility through a patient-centered approach. Accordingly, a Patient Advisory Council dedicated to the XLHED program has been established and provides the opportunity to Esperare and patient groups representatives to work in close collaboration. In particular, clinical development plans are discussed in order to refine development and generate robust and necessary data for an application and to contribute to the common goal of giving the greatest chance of success to this program, with the ultimate objective of maximizing the benefits and minimizing the risks for XLHED patients and their families. Requests for compassionate use/expanded access will be considered on a case by case basis , in accordance with applicable regulations and in alignment with our mission and values and with the safety of the patient and childbearing mother as a priority. Of note, ER-004 is delivered to the patient before birth by injection into the amniotic fluid, an untried route of administration that requires careful consideration in each individual case. Inquiries regarding the expanded access may be sent to info.er004@esperare.org . Esperare will endeavor to acknowledge receipt of any expanded access questions or requests within 5 business days. Available Therapies via Single-Patient EA ER-004 (also known as EDI200) is a protein replacement therapy designed as a substitute for endogenous EDA, a protein missing in XLHED .
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