The first licensed hematopoietic progenitor cells-cord cell therapy has received FDA approval. This is the first approval of a license application for cord blood.
Hemacord (New York Blood Center) is indicated for use in hematopoietic stem cell transplantation procedures in patients with disorders affecting the hematopoietic system.
"The use of cord blood hematopoietic progenitor cell therapy offers potentially life-saving treatment options for patients with these types of disorders," Karen Midthun, MD, director of the FDA's Center for Biologics Evaluation and Research, said in the press release.
Hemacord contains hematopoietic progenitor cells from human cord blood.
In 2009, the FDA issued the "Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications" to help manufacturers apply for licensure for certain cord blood units. FDA instituted a 2-year phase-in period for HPC-C manufacturers to submit either a license application or an investigational new drug application. The phase-in period ended Oct. 20, 2011, and these manufacturers now must submit such applications, according to the press release.
Approval was based on reliance on safety and efficacy data submitted to a public docket and data submitted in the license application that demonstrated compliance with other regulatory requirements.
The treatment includes a boxed warning of the risks for graft-versus-host disease, engraftment syndrome, graft failure and infusion reactions. Patients receiving treatment should be monitored carefully. In addition, a risk-benefit assessment, unit selection and administration of Hemacord should be conducted under the direction of a physician experienced in hematopoietic stem cell transplantation.
The licensing of Hemacord, banked umbilical cord blood
for unrelated allogeneic transplantation manufactured by the National Cord
Blood Program (NCBP) at the New York Blood Center, marks an important milestone
in the journey of bringing umbilical cord blood, a source of hematopoietic stem
and progenitor cells, to licensure. It is anticipated that several more public
cord blood banks will obtain licenses over the next few years. Although the
license initially was viewed as a way to improve the safety, purity, potency
and quality of cord blood units used for transplantation, these goals have not
yet been realized. Many cord blood units — including those at the NCBP,
and those at other banks and international units — will never qualify for
licensure.
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Joanne Kurtzberg
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Yet, cord blood transplantation has been successfully
used in clinical practice for more than 20 years using these unlicensed units.
Early on, it was demonstrated that cord blood donors did not have to match as
closely as adult bone marrow donors, which allowed cord blood to provide
increased access to transplantation for patients unable to find a fully matched
unrelated donor. Currently, there are more than 140 banks worldwide and an
international inventory of approximately 800,000 unrelated donor units. More
than 30,000 transplants have been performed with these unlicensed units for
standard clinical transplant indications over these 2 decades, showing results
that are similar to other hematopoietic stem cells sources like bone marrow or
mobilized adult blood. With the implementation of licensure, access to
unlicensed cord blood units (the vast majority at the present time) must now be
managed under investigational new drug applications. This has created increased
regulatory burden for banks and transplant centers alike. While licensure has
increased the cost of cord blood units, it remains to be seen whether it will
increase quality. If standards applied to cord blood were applied to bone
marrow or peripheral blood progenitor cells, there is a great risk that the
provision of these donors might become cost prohibitive, threatening the field
of unrelated donor transplantation overall. The FDA is applying standards and
regulations created for drug manufacturing to cord blood. This results in
frequent disconnects because manufacturing cells is not the same as
manufacturing drugs. As licensing proceeds, it would be wonderful if the FDA
could create appropriate standards and regulations for manufacturing of cells
that would maintain and improve quality without increasing costs. This strategy
would benefit patients and allow this life-saving therapy to be provided in a
challenging health care environment.
– Joanne Kurtzberg, MD
Director of the Pediatric Blood and Marrow Transplant
Program
Duke University Medical Center, Durham, N.C.
Disclosure: Dr. Kurtzberg reports no relevant
financial disclosures.