FDA Pushes Risk-Based Approach to Nanomaterial Drugs

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By Greg Langlois

Developers of drugs containing nanomaterials—substances so small they can’t be seen with a regular microscope—got some advice on how to assess their risks in FDA draft guidance issued Dec. 15.

Drug products containing nanomaterials “may merit particular examination” because their attributes can differ from those without those substances, the Food and Drug Administration explained in the draft. Nanomaterials—measured in nanometers, or one-billionth of a meter—can serve a variety of functions, such as being an active ingredient or a carrier of one.

Nanomaterials’ minuscule size, allowing them to penetrate body structures that larger substances may not, has raised fears they can be toxic or cause environmental problems.

Without having read it in detail yet, Lynn Bergeson, managing partner of Bergeson & Campbell PC, told Bloomberg BNA in a Nov. 15 email the FDA “has done its characteristic good job of providing useful and user-friendly information to stakeholders innovating and manufacturing drug and biological products containing nanomaterials.”

“The [g]uidance provides significant granularity on technical and scientific issues, including nanomaterial characterization methods, [absorption, distribution, metabolism, and excretion] considerations, risk considerations for specific routes of exposure, among other topics,” said Bergeson, whose firm includes a nanotechnology practice. The document “would appear to join a long line of useful FDA guidance documents around the issue of nanotechnology and the use of nanomaterials in FDA[-]regulated products.”

No Pre-Judgment

The agency issued final guidance in 2014 explaining how FDA-regulated product developers should determine whether a product applies nanotechnology. This new draft guidance on risk assessment should be read in concert with that, it said.

In both documents and in public statements, the FDA said it doesn’t judge all products containing nanomaterials or involving the use of nanotechnology as “intrinsically benign or harmful.”

“Rather, for all products (nanotechnology-derived or otherwise), FDA considers the characteristics of the product and its safety and effectiveness for use,” the agency said in the draft guidance.

The agency added it doesn’t presuppose how it would assess any particular nanomaterial-containing drug product. Instead, safety, effectiveness, health impact, and regulatory status of such products “are currently addressed on a case-by-case basis using FDA’s existing review processes,” the draft said.

“FDA’s customary caveats,” Bergeson said, “are noted in the guidance, reassuring stakeholders that these important bedrock principles are unchanged.”

The FDA also may in the future develop specific guidance on commonly used types of nanomaterials, it said, such as liposomes. The agency has approved numerous liposome-based drug products, including the Onivyde (irinotecan) pancreatic cancer chemotherapy drug, which Merrimack Pharmaceuticals Inc. sold to Ipsen SA this year; and Janssen Products LP’s DOXIL (doxorubicin), for treating multiple myeloma, ovarian cancer, and AIDS-related Kaposi’s sarcoma.

Risks to Consider

The draft guidance describes a risk-based approach to nanomaterial-containing drug development and lists a number of factors developers should consider, including the need for an adequate description of included nanomaterials, such as their chemical composition, size, charge, and function; physical and chemical stability; route of administration; and distribution, biodegradation, and accumulation within the body.

Developers should also address the maturity of the nanotechnology applied, including manufacturing and analytical methods, the draft said.

The draft also addresses nanomaterials’ potential effect on the environment. Federal law requires agencies to assess the environmental impact of their actions, and the FDA requires product applicants to submit an environmental assessment or assert whether their products can be excluded from environmental review.

“In light of the current, evolving state of scientific knowledge regarding the impact of nanomaterials in the environment,” the FDA said, it will use a “case-by-case approach” to assess whether drug products containing nanomaterials qualify for exclusion or require an environmental assessment.

Drug developers should notify the FDA early on if they intend to claim an exception or submit an environmental assessment, the FDA said.

To contact the reporter on this story: Greg Langlois in Washington at glanglois@bloomberglaw.com

To contact the editor responsible for this story: Randy Kubetin at rkubetin@bloomberglaw.com

For More Information

The draft guidance is available at http://src.bna.com/u0D.

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