FDA Acts on Latest Childhood Cancer Drug Shortage

by Kathleen Ruddy, CEO, St. Baldrick's Foundation
February 7, 2014

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Last November, we reported on a shortage of the generic, sterile injectable chemotherapy drug, daunorubicin, that is used to treat children with acute myeloid leukemia (AML) as well as acute lymphoblastic leukemia (ALL).

Pediatric oncologists have depended on daunorubicin  as a go-to treatment for kids with these leukemias for nearly 40 years.  The fact that it has been difficult for many hospitals to obtain caused alarm bells to go off within the pediatric cancer community and sparked an inquiry from Congress to the U.S. Food and Drug Administration (FDA).

Now, roughly two months after the FDA received a letter about the shortage of daunorubicin from Rep. Michael McCaul (R-Texas) and Rep. Chris Van Hollen (D-Md.), co-chairs of the Congressional Childhood Cancer Caucus, the agency has replied.

According to the FDA, the daunorubicin shortage has two primary causes:  1) Only two U.S. companies manufacture the drug, and one of them — Ben Venue Labs of Bedford, Ohio — went out of business in late 2013, and 2) The sole remaining U.S. manufacturer — Teva Pharmaceuticals of North Wales, Pennsylvania — announced a product recall due to “potential presence” of “particulate matter” in the drug.

Taken together, these two events created a perfect storm to significantly limit supplies of daunorubicin in the U.S.

But, according to the FDA, Teva has daunorubicin on hand and is executing a supply recovery plan designed to ensure that  patients who need the drug are able to get it. The plan includes posting a toll-free phone number on the FDA website for patients and health care professionals to call who most urgently need the drug. All orders will be shipped directly from Teva to bypass wholesalers, distributers, and other middlemen who could exploit the shortage for financial gain by stockpiling supplies. Teva expects a limited supply of the drug through June 2014.

The FDA also says it is in regular contact with Teva over this issue, and is exploring whether alternative sources of  daunorubicin may be available. The FDA letter concludes with a list of seven tools that the agency can use if the daunorubicin shortage continues, or to address other drug shortages in the future.

In other news related to addressing drug shortages, Congress passed a law late last year intended to make it easier for specialty pharmacies to custom make generic medicines that are in short supply. Known as compounding pharmacies, the new law allows these operations to register with the FDA for the first time.  In exchange, compounding pharmacies can market their services more broadly and potentially fill the void when drug product is in short supply due to manufacturing issues.

While the exact number of compounding pharmacies in the U.S. has not been published, it is estimated that there are at least several hundred nationwide. Encouragingly, 14 compounding pharmacies have already registered with FDA as of mid-January. Hopefully, this will eventually help kids with cancer and their families who rely on daunorubicin and other generic drugs that can fall into short supply, seemingly overnight.

We thank Speak Up advocates for acting quickly on this issue last fall. Your involvement does make a difference. Spread the news of your success by sharing information about this issue and Speak Up for Kids’ Cancer with your friends and family.

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