Our Commitment to Patients

At Liquidia, we’re dedicated to improving the lives of people living with serious and rare lung diseases.  Our mission is to deliver innovative therapies safely and efficiently through clinical research and FDA approval.

We understand that some patients may not be able to join a clinical trial. In certain cases, patients and their doctors may request access to a Liquidia investigational therapy through a program called Expanded Access (sometimes called Compassionate Use).

 

What Is Expanded Access?

Expanded Access allows a patient with a serious or life-threatening illness to receive an investigational therapy — a medicine still being studied and not yet approved by the U.S. Food and Drug Administration (FDA) — outside of a clinical trial.

An Expanded Access request may be considered when:

  • The patient has a serious or immediately life-threatening condition.
  • There are no satisfactory approved treatment options available.
  • The patient cannot join or access an ongoing clinical trial.
  • There is evidence that the investigational therapy may help the patient.
  • Providing the therapy will not interfere with ongoing clinical trials.

 

Who Can Request Expanded Access?

Requests must come from the patient’s treating physician. The physician will:

  • Review the potential risks and benefits with the patient.
  • Contact Liquidia directly to discuss the possibility of Expanded Access.
  • Work with the FDA and an Institutional Review Board (IRB) if access is approved.

 

How to Request Access

If you are a physician seeking Expanded Access for a patient, please contact us:

Email: expandedaccess@liquidia.com
Phone: +1-888-530-9216

Liquidia will confirm receipt of the request within five (5) business days.

Please include:

  • The patient’s diagnosis and medical history
  • Explanation of why other treatments are not suitable
  • The rationale for potential benefit
  • The proposed dosing and monitoring plan

 

How We Review Requests

Each request is carefully reviewed by Liquidia’s medical and regulatory teams. We consider:

  • The scientific data available for the investigational therapy
  • The patient’s medical condition
  • The potential benefits and risks
  • The availability of the investigational therapy
  • Any impact on ongoing or planned clinical trials

Approval requires both Liquidia’s agreement and FDA authorization through an individual patient IND or other applicable mechanism.

 

If Access Is Granted

If Liquidia and the FDA approve Expanded Access:

  • The treating physician must obtain FDA and IRB approvals.
  • The patient (or caregiver) must provide informed consent in the form approved by Liquidia Compliance.
  • The physician is responsible for monitoring the patient’s response and reporting safety information to Liquidia and the FDA.
  • Before deciding whether to participate in Liquidia’s Expanded Access Program, physicians and patients should carefully consider the risks associated with using an investigational product.

 

Costs

Liquidia may provide the investigational therapy at no cost or may charge only the direct costs related to providing it, as permitted by FDA regulations (21 CFR 312.8).

 

Clinical Trials Are Still the Best Option

Clinical trials are the best way to access investigational medicines and to help advance research for all patients. To learn more about ongoing Liquidia studies, visit ClinicalTrials.gov and search “Liquidia”

 

Program Closure Upon FDA Approval

Upon approval of the investigational product by the FDA, Liquidia will work closely with the treating physician and patient to ensure a seamless transition once the commercial product is available on the market for the specific disease.

 

This policy is published in accordance with the requirements of the 21st Century Cures Act. Liquidia reserves the right to revise this policy as new clinical information, regulatory guidance, or therapies become available.                                                                                                                                             
Last updated: September 2026