Approximately 30% of IND applications are placed on hold due to CMC issues. Don’t let this happen to you.

Streamline the Time to Submission and Approval or Clearance

From the early stages of product development, through to market authorization, Akron works closely with clients to develop effective regulatory drug development strategies for advanced biological therapies.  Akron has extensive knowledge and experience in U.S. and international regulatory affairs, with a proven track record in achieving approvals.

Regulatory Framework

All clinical applications and devices involving stem cells are regulated at some level. In most countries, if not all, the use of minimally manipulated autologous stem cell implantation is regulated by the local or hospital ethics committee, or both. Exempt from FDA regulation are vascularized human organs for transplantation, autologous products reimplanted during the same surgical procedure, minimally manipulated marrow for homologous use, or reproductive cells implanted into the partner of the donor.

Changes made to 21 CFR 1271 in 2004 regarded “more than minimally manipulated” autologous human cells as an Investigational New Drug (IND) requiring a Biologics License Application. As a result, only limited procedures can be performed with stem cells in the clinical setting.  The goals of regulation are to prevent unwitting use of contaminated tissues that have the potential for transmitting infectious diseases, and to prevent improper handling or processing that might contaminate, damage, or change the properties of the agent.

Stem Cell Drug Substance is Special

Unlike small-molecule pharmaceuticals, cells and stem cells are living entities that cannot be chemically synthesized. Therefore, unique considerations must be given to the donor and the source of the cells, and to subsequent processing.  The most significant difference between small-molecule pharmaceutical development and stem cells is the definition of Drug Substance (DS).  Drug Substance means an active ingredient that is intended to furnish pharmacological activity.

If  the DS is a stem cell or cell, the DS includes intermediates that are used in the synthesis of such an ingredient. In practice, this means that any reagent or agent coming into contact with the cell during the product development lifecycle is considered the drug substance.  For this reason, it is important to work closely with Akron from the beginning, and to define your needs and goals and the reagents you will be using.

Regulatory Strategy, Filing, and Support

Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) are defined as articles containing or consisting of human cells or tissues that are intended for implantation, transplantation, infusion, or transfer to a human recipient, as outlined in 21 CFR 1271.3 d.  If your product will be regulated as an investigational new drug, Akron can support your application before you file the IND.  Akron will do so by executing and compiling necessary information, and by providing

  • Strategic support and scientific rationale
  • A well-developed, controlled manufacturing plan
  • Data on product characterization
  • Data to support specifications for the product’s quality control and release