Are they drugs or organs?

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A group of more than 50 experts in the field of pancreatic islet cell transplantation calls for the reclassification of the type 1 diabetes biological drug therapy to human organ.

On April 15, an advisory panel to the U.S. Food and Drug Administration (FDA) voted to support approval of an application for a biological license (BLA) for the allogeneic product of human islets donislecel (Lantidra) developed by CellTrans.

The treatment would be aimed at people with type 1 diabetes complicated by hypoglycemic ignorance or other problems that lead to extreme glycemic lability while optimized insulin therapy.

During the public comment period of the one-day meeting, Piotr Witkowski, MD, PhD, director of the islet pancreatic cell transplant program at the University of Chicago, Illinois, he spoke against FDA approval on behalf of the Islets for US collaboration. Comprised of more than 50 experts in the field, the organization aims to “protect” [islets] of the marketing and preservation of the human pancreas and islets as public resources for the public good “.

Witkowski argued that instead of the FDA granting the company an exclusive BLA, human islets should be regulated as transplanted organs under the auspices of the Health Resources and Services Administration (HRSA) under the supervision of the Organ contracting and transplantation network (OPTN) i United Network for Organ Sharing (SOME).

The FDA’s stance means that islet transplants are behind other U.S. states

Human pancreatic islet cells are isolated from the pancreas of dead donors and transplanted into the recipient’s liver. They require the same immunosuppressive regimens as all organ transplants.

“Islets are human microorganisms and should be regulated like the pancreas and other human organs … Islets are not drugs and they are not cell therapy,” Witkowski stressed.

He noted that many parts of the world, including countries in Europe, Canada, Australia and Japan, regulate islets as organs rather than drugs. “The FDA’s position that allogeneic islets are drugs and require a BLA has prevented islet transplantation from becoming a standard U.S. care procedure, in contrast to many other countries,” he noted. .

Witkowski argued that many American academic transplant centers have successfully processed human islets for transplantation in BLA-free clinical trials in the last 20 years, but unlike one manufacturer, these institutions are not in a position to obtain this. license.

He gave seven years of marketing exclusivity, he said, that the manufacturer would give him leverage on the availability, cost and safety control of patients after the transplant.

According to HRSA standards, the OPTN / UNOS oversees organ transplant programs and monitors patient outcomes after transplantation, but this control is not required for medications. This raises a security concern, he said.

FDA, CellTrans see it differently

There were no responses to public comment comments during the protocol meeting, therefore Medscape Medical News contacted the FDA, CellTrans, and the U.S. Department of Health and Human Services (HHS). (Both the FDA and HRSA are agencies within HHS and OPTN is operated by UNOS under contract with HHS).

An FDA spokesman responded by email: “The manufacture and transfer of allogeneic cells from the pancreatic islets is not analogous to whole organs. pancreas transplant, and we do not agree with the suggestion that pancreatic islets meet the definition of a vascularized human organ. “

The spokesman also noted: “The FDA established regulatory requirements for allogeneic islets based on the specific characteristics of this product, its manufacture and intended use, and did so in a transparent manner with the contributions of the relevant stakeholders. There is no plan to review or reconsider this regulatory approach “.

And CellTrans said, “CellTrans introduced a BLA following federal regulations governing cell therapies. The code of federal regulations on biological products aims to ensure safe, high-quality therapies. CellTrans’s commitment to safety it is evident in the files it has filed with the FDA and will continue to fulfill all government mandates to maintain safety. “

The company also said in its email that it “intends to offer islet cell transplantation to patients who need it as a standard care option. As part of its commitment to facilitate such access, CellTrans has given up all the exclusive rights at your disposal as part of your application. “

In addition, “CellTrans is an islet manufacturer and is not involved in patient care or organ allocation. CellTrans simply processes organs accepted by the transplant center using the UNOS allocation system and provides the preparation of the islet cells in the hospital transplant center “.

The HHS press office did not respond on time or after repeated subsequent requests.

According to the discussion at the advisory group meeting, it is unclear how many patients will be affected by this problem.

The members of the group weighed the risks of immunosuppression against the benefits of transplanted islets, as in recent years advances in technology and treatment of diabetes have reduced the number of patients with hypoglycemic ignorance and extreme glycemic lability.

The FDA is expected to announce a decision on licensing in August 2021.

Witkowski tea has not reported any relevant financial relationships.

Miriam E. Tucker is a freelance journalist based in the Washington, DC area. She is a regular contributor to Medscape, with other work appearing in the Washington Post, NPR’s Shots blog, and Diabetes Forecast magazine. He is on Twitter: @MiriamETucker.

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