Expanded Access

1) Purpose

BioMendics, LLC is developing TolaSure for Epidermolysis Bullosa Simplex (EBS). This policy explains how physicians may request treatment use of TolaSure outside a clinical trial, the criteria we use to evaluate requests, and our commitments to patients, investigators, and regulators. FDA’s Expanded Access (EA) regulations (21 CFR 312 subpart I) permit use of an investigational drug for treatment when specific criteria are met.1

2) Background on Breakthrough Therapy Designation

TolaSure has received Fast Track Designation (FTD) from FDA, an expedited development/review program for drugs that facilitates the development and potentially expedites the review of drugs intended to treat serious or life-threatening conditions and fill an unmet medical need. FTD does not guarantee expanded access (EA) or alter the statutory EA criteria, but it can facilitate scientific and regulatory interactions during development.2

Because TolaSure remains under clinical investigation, available efficacy and safety information is limited. EA decisions will be based on the totality of evidence available at the time of review.

Clinical Trial of Record: NCT#07027345 and link to clinicaltrials.gov study page.

3) Policy Statement

BioMendics, LLC supports EA when:

  • The patient has a serious or immediately life-threatening disease and no comparable or satisfactory alternatives exist;
  • Potential benefit justifies the potential risks and those risks are not unreasonable in the treatment context; and
  • EA will not interfere with the initiation, conduct, or completion of clinical trials or the overall drug-development program.1

Clinical trials remain the preferred route of access. If a patient is eligible for an active trial and can reasonably enroll, we will prioritize trial participation over EA.2

4) Types of Expanded Access We May Support1

  • Single-patient EA (including emergency use) under 21 CFR 312.310
  • Intermediate-size patient population under 21 CFR 312.315
  • Treatment protocol / Treatment IND under 21 CFR 312.320

5) Eligibility & Evaluation Criteria

Expanded Access requests are reviewed by appropriate BioMendics personnel, which may include representatives from Clinical Development, Medical Affairs, Regulatory Affairs, Pharmacovigilance, Manufacturing, and Executive Management.

When evaluating requests, we consider:

  1. Medical Criteria
    • Diagnosis, severity, and prognosis; lack of satisfactory alternatives; prior therapies and response; concomitant medications; known risks of BM-3103; and whether the anticipated benefit outweighs potential risks.1

Pediatric patients: Requests involving pediatric patients will be evaluated using the same criteria described in this policy, with additional consideration of age-specific safety information, dosing considerations, developmental factors, and monitoring requirements.

  1. Development Integrity
    • Whether EA could compromise ongoing or planned trials, including enrollment, interpretation of efficacy/safety, or regulatory timelines.1
  2. Supply & Feasibility
    • Adequacy of manufacturing and qualified clinical supply, appropriate dosage form/strength, stability, labeling, and ability to monitor and report safety.
    • In the event of limited product supply, BioMendics may prioritize product allocation to ongoing clinical trials and regulatory development activities.
  3. Regulatory & Ethical Requirements
  • Ability to obtain FDA authorization, IRB review or concurrence as applicable, informed consent, and compliance with applicable regulatory requirements.

Submission of a request does not guarantee that EA will be granted. Each request is evaluated individually based on available evidence, patient circumstances, product supply, and development considerations.

If a request is denied, we will communicate the primary reason (e.g., eligible for a trial, insufficient safety data for the population, insufficient supply, or inability to ensure monitoring).

6) How Physicians Request Expanded Access

All requests must be submitted by a licensed treating physician who is willing to assume responsibility as the sponsor-investigator or investigator of record and who can comply with applicable FDA, IRB, safety reporting, and recordkeeping requirements.

For Non-Emergency Single-Patient Requests:

Email info@biomendics.com and copy kmcguire@biomendics.com to obtain the current Expanded Access (EA) request package.

  1. Submit the following to BioMendics, LLC: brief medical history, diagnosis, prior therapies, proposed dosing/duration, monitoring plan, investigator/site qualifications, and statement of unavailability of alternatives including ongoing clinical trials.
  2. If preliminarily acceptable, BioMendics will issue a Letter of Authorization (LOA) for cross-referencing the BioMendics IND (if applicable) or provide a company-sponsored treatment protocol.
  3. Company agreement to provide TolaSure does not constitute FDA authorization. Treatment may not begin until all applicable regulatory and ethical approvals are obtained, except as permitted under emergency expanded access provisions.
  4. The treating physician submits Form FDA 3926 (or appropriate IND submission) to FDA and obtains IRB approval prior to treatment (unless emergency; see below).
  5. BioMendics will acknowledge receipt of a complete Expanded Access (EA) request within 2–3 business days. Subject to medical urgency and the completeness of the submission, BioMendics will review the request and generally provide a decision to the treating physician, along with any applicable next steps, within 10 business days (approximately 2 calendar weeks).

Emergency Use:

The physician should contact FDA’s Emergency Call Center (866-300-4374) for telephone authorization before treatment, when possible. IRB notification within 5 business days and Form FDA 3926 within 15 business days are typically required.

BioMendics, LLC aims to review complete non-emergency EA requests promptly and will communicate decisions and next steps to the physician.

7) Physician Responsibilities (upon approval)

  • Obtain FDA authorization and IRB approval (or follow emergency procedures noted above).
  • Obtain informed consent from the patient/legal representative.
  • Ensure appropriate site capabilities for drug handling, administration, and patient monitoring.
  • Maintain drug accountability; adhere to the treatment plan provided by BioMendics .
  • Report adverse events and other safety information per 21 CFR 312.32 and company instructions
  • Return or destroy unused drug per instructions.

8) Company Responsibilities

  • Provide scientific information to support the request; supply investigational product if available and appropriate; and support regulatory interactions as feasible.
  • Maintain pharmacovigilance and submit safety reports to FDA as required.
  • Monitor EA use to ensure it does not compromise clinical development.
  • BioMendics reserves the right to discontinue treatment if new safety information emerges, product supply becomes unavailable, regulatory authorities require discontinuation, or continued treatment is no longer considered medically appropriate.

9) Cost Recovery

BioMendics may provide TolaSure, 5% w/w (BM-3103) at no charge or may seek FDA authorization to recover direct costs consistent with 21 CFR 312.8 (e.g., drug manufacturing, shipping, handling) when EA is permitted. Any charging will comply with FDA’s charging rules and guidance.

10) Data Use & Privacy

Clinical information generated during Expanded Access is not a substitute for adequate and well-controlled clinical trials. BioMendics may collect and evaluate safety information arising from Expanded Access use. Such information may be included in regulatory safety reporting and may contribute to the overall assessment of the investigational product's benefit-risk profile. Patient privacy will be protected consistent with applicable law, informed consent requirements, and IRB requirements.

11) Transparency & How to Contact Us

This policy will be posted on BioMendics’ website: www.biomendics.com
EA Requests & Questions: info@biomendics.com and copy kmcguire@biomendics.com
After-hours emergencies: Please contact FDA’s Emergency Call Center (866-300-4374) noted above and non-emergency email druginfo@fda.hhs.gov with “Emergency EA” in the subject line.

12) Geographic Scope

This policy addresses U.S. requests. For requests outside the U.S., clinicians should follow their national frameworks (e.g., EU compassionate use) and contact BioMendics at info@biomendics.com and copy kmcguire@biomendics.com for region-specific procedures.

13) Amendments & Discontinuation

BioMendics may revise, suspend, or terminate this policy at any time (e.g., due to safety findings, supply constraints, or regulatory changes) or upon FDA approval and commercial availability of TolaSure, 5% w/w (BM-3103).

Appendix A — Quick Checklist for Treating Physicians

  • Patient has a serious or life-threatening disease and no satisfactory alternatives.
  • Anticipated benefit justifies the risk; appropriate monitoring is feasible.
  • Patient is not eligible for, or cannot access, an active clinical trial.
  • IRB engaged; FDA authorization (Form FDA 3926 for single-patient) planned.
  • Drug handling & accountability in place; safety reporting understood.
  • 21 CFR part 312, subpart I — Expanded Access (requirements; individual, intermediate, and treatment uses).

Key References

  1. FDA Guidance (Nov 2022): Expanded Access to Investigational Drugs for Treatment Use — Q&A.
  2. 21 CFR 312.8 & FDA Charging Guidance (2024) — Charging for investigational drugs, including EA.
  3. FDA BTD FAQs (2024) & 21 U.S.C. §356 — Breakthrough Therapy Designation framework.
  4. FDA “How to Request Single-Patient EA” (2025) — Practical steps & Form FDA 3926.
  5. FDA Expanded Access page (2025) — Emergency contact information.