Medical Affairs

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Medical Information

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If you have a Medical Information question regarding a Zevra product or would like to request a meeting with your local Medical Science Liaison, please contact our Medical Information team at:

T: 1-844-600-2237
Email: [email protected]

To report a suspected side effect, adverse reaction, or concern about the quality of a Zevra product, contact us at toll-free phone 1-844-600-2237.

Live assistance available Monday-Friday, 9am-5:30pm EST

Adverse events may also be reported to the FDA's MedWatch Program at www.fda.gov/medwatch or by calling (800) FDA-1088.

Investigator-Initiated Studies

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The Zevra Therapeutics Investigator-Initiated Studies (IIS) Program will accept unsolicited submissions within our current areas of interest as noted below. Submissions from qualified investigators (i.e., health care professionals, scientists, and researchers) will be reviewed by the Zevra Investigator and Collaborative Research Review Committee for alignment with the AOIs. Research submissions that are not complete, that are not within the scope of these AOIs, or have low relevance to the therapeutic area, may be rejected without further review.

Proposals will be evaluated on their scientific merit to advance science, clinical medicine, and patient care according to availability of resources. Support can include, but is not limited to, drug material, expert advice and/or funding to pay expenses directly related to the research based on fair market value.

The investigator, or the institution the investigator is affiliated with, will serve as the trial sponsor. As such, the sponsor assumes responsibility for initiating and conducting the trial, directing the administration of the trial drug, ensuring compliance with all local laws and regulatory requirements, and analyzing and communicating all trial results.

The goal of the IIS is to advance science and contribute to the development of better medicines for patients consistent with the company’s overall research and development strategies.

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Areas of Interest

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MIPLYFFA

  • Further understand the mechanism of action of arimoclomol
  • Further understand the benefit risk of arimoclomol for NPC
  • Clinical or pre-clinical evidence of the effectiveness and safety of arimoclomol in neurological or other lysosomal storage disorders

IIS Submission Process

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The submission process consists of 2 key steps:

  1. Concept review
    1. Review of a 1-2 page summary of the trial rationale, objectives, and design
    2. A description of the type of support requested (e.g., funding, trial drug)
    3. An estimated budget (if funding is requested)
  2. Full proposal review

If the concept is approved for further review, you will be invited to submit a full proposal application including:

  • Detailed trial protocol
  • Comprehensive budget
  • Additional supporting documents as needed

To inquire about submitting a proposal, please send an email to [email protected]