GMP Peptide Manufacturing for Emergency INDs (EINDs)

An EIND can be a patient’s lifeline. This article will examine the Emergency IND (EIND) process under the Expanded Access program and the pivotal role that GMP Peptide contract development and manufacturing organizations (CDMOs) play, in meeting the stringent demands of sponsors and investigators.
In the realm of drug development, clinical trials remain the cornerstone for evaluating the safety and efficacy of investigational new drugs. To begin a clinical investigation into human subjects in the U.S., an IND (Investigational New Drug) application must be submitted to the FDA. It is through clinical trial enrollment that a patient is able to access an experimental drug.
Unfortunately, some individuals who may be critically ill, with no viable therapeutic options, will not qualify for clinical trial enrollment. In such cases, the FDA offers Expanded Access (EA) or “compassionate use” IND programs. These INDs are intended primarily for treatment rather than data collection on drug safety and efficacy. Of these alternate INDs, the EIND, a single-use Emergency IND is a pathway to enable patients facing life-threatening conditions access to investigational drugs.
Expanded Access (EA) to Investigational Peptide Drugs
There are three categories of Expanded Access programs applicable to experimental Peptide drugs:
- Expanded access for individual patients, including emergency situations, requires a "treatment-use" IND specific to each patient. This category will be the primary focus of our discussion.
- Expanded access for intermediate-sized groups, conducted under a protocol submitted by the sponsor to an existing IND.
- Expanded access for broader treatment use in larger populations, also managed under a protocol submitted to the existing IND. These programs are often utilized to provide continued access to investigational drugs for clinical trial participants while awaiting FDA approval.
Emergency Use IND Eligibility
For a patient to qualify for an Emergency IND, they must meet the three criteria required for any Expanded Access program, plus a fourth emergency criteria:
- The patient must have a serious or immediately life-threatening disease or condition with no satisfactory alternative therapy available.
- The potential benefits must outweigh the potential risks of the treatment.
- The treatment must not interfere with or jeopardize the drug development, such as by depleting drug needed for clinical trials.
-
In addition to the eligibility requirements listed above for Expanded Access, there exists an urgency to act quickly on the patients behalf. Due to emergency circumstances, insufficient time would be available for IRB (investigational review board) review and approval of the IND/EIND. The EIND offers to individual patients and their healthcare providers, access to investigational drugs during an emergency situation for immediate treatment of the patient.
In most EIND emergency cases, the patient’s physician serves as both the EIND sponsor and investigator, who is responsible for working with the original IND sponsor for materials, handling the application process and supervising the administration of the treatment.
Key Factors to Consider when choosing a CDMO for EIND Peptides
The FDA mandates that investigational new drugs (INDs) be manufactured under Good Manufacturing Practice (GMP) standards to help reduce risks to patient safety. For EIND applications, these requirements are even more critical, as the drug will be administered under urgent and potentially high-risk circumstances.
Compliance with Regulatory Standards
The CDMO must operate under a Quality Management System to ensure maintenance of proper documentation for strict adherence to manufacturing protocols, and rigorous in-house QA/QC. The CDMO should also offer a Chemistry, Manufacturing, and Controls (CMC) package required for IND submission.
Reliability
The CDMO should have a long history of experience , manufacturing simple to highly complex peptides and peptidomimetics, at a range of scales. The production facility should ideally be based in the US and offer segregated upstream and downstream processing areas for complete process control.
Speed
Sponsors should partner with Contract Development and Manufacturing Organizations (CDMOs) that can swiftly deliver GMP-grade peptide batches. Uninterrupted access to high-quality drug substances for both clinical trials and emergency use, is essential if the need arises.
AnaSpec: A Leader in Fast GMP Peptide Manufacturing
AnaSpec specializes in delivering custom peptide API drug substance, API excipients, and precursors that comply with GMP standards while meeting urgent timelines. The company’s ability to balance speed with quality is vital to ensuring that patients receive life-saving therapies without unnecessary delays.
- Speed Without Compromise: AnaSpec’s streamlined processes enable rapid production without sacrificing compliance or quality. In cases where sponsors/investigators require additional drug substance quickly, AnaSpec is equipped to accommodate requests with fast lead-times.
- Tailored Solutions: Our peptide synthesis services are designed with flexibility to address the specific needs of each sponsor and investigator, particularly in emergency clinical situations.
Conclusion
Emergency INDs provide a critical avenue for patients facing life-threatening conditions to access investigational therapies when no other options exist. This pathway exemplifies the delicate balance between urgency, safety, and regulatory compliance, ensuring that patients in dire need receive timely treatment without compromising ongoing drug development programs.
In this process, the importance of GMP peptide manufacturing cannot be overstated. The ability to produce high-quality, reliable, and regulatory-compliant peptides under tight timelines is essential to meeting the demands of IND/EIND applications. Partnering with a trusted CDMO, like AnaSpec, ensures that sponsors and investigators can confidently deliver life-saving therapies while adhering to FDA requirements.
By leveraging expertise in GMP peptide manufacturing, innovators and healthcare providers can accelerate the delivery of emergency treatments, ultimately bringing hope and critical care to patients when they need it most.
Disclaimer: This article is intended for informational purposes only and should not be considered as legal, medical, or regulatory advice. For the most accurate and up-to-date information on IND guidelines and regulations, please consult the official FDA website and/or FDA offices.
References
-
U.S. Food and Drug Administration. (n.d.). Expanded access: How to submit a request (Forms). Retrieved December 3, 2024, from https://www.fda.gov/news-events/expanded-access/expanded-access-how-submit-request-forms#Summary
U.S. Food and Drug Administration. (n.d.). Emergency use of an investigational drug or biologic. Retrieved December 3, 2024, from https://www.fda.gov/regulatory-information/search-fda-guidance-documents/emergency-use-investigational-drug-or-biologic