Update March 8, 2015: On March 6th the FDA approved this drug for sale in the U.S. with a new brand name, Zarxio.
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There’s big news from the FDA today with implications not just for the pharmaceutical industry, but for patients. The U.S. agency’s ODAC unanimously recommended* approval for Sandoz’s version of filgrastim (Zarzio, EP2006,
Approval of a biosimilar drug like filgrastim is a big deal, first because U.S. oncologists prescribe this to cancer patients all the time. It comes in vials and pre-filled syringes. Because Neupogen (the old drug) costs over $300 per dose, copays can be significant and are potentially limiting.
Cancer patients typically take filgrastim daily for a period during some or all cycles of chemotherapy. Most often, it’s prescribed for the “nadir” part of the treatment cycle – when the white blood cells run low, and patients are most vulnerable to severe infection. The drug is also given to patients in preparation or after bone marrow transplantation, sometimes by intravenous injection, and for some rare blood conditions.
Zarzio® (EP2006, filgrastim) and two other biosimilar drugs like Neupogen have been used in Europe since 2008, reviewed here. Last month, investigators reported on results of a Phase III, randomized trial comparing Zarzio and Neupogen in breast cancer patients receiving chemotherapy. The PIONEER study results were presented last month at the annual meeting of the American Society of Hematology. The main finding of the Sandoz-sponsored trial is that the drugs have indistinguishable clinical activity.
This approval matters because either drug could be used in patients receiving not just one particular chemotherapy, but many kinds and combinations of chemotherapy for all sorts of cancers. Filgrastim works by boosting production of infection-fighting white cells in the bone marrow. A well-known side effect of cancer treatment is that the white cells tend to drop during chemotherapy. Since the development of filgrastim, this potentially lethal complication has become far less frequent.
If the FDA’s decision leads to increased access to biosimilar drugs for patients with other conditions, like rheumatoid arthritis, Crohn’s disease, lupus and a host of other diseases for which monoclonal antibodies and other biological agents might be prescribed, that would be huge. The caveat, of course is needed, careful testing for these kinds of complex pharmaceuticals – that they are “clean,” equally safe and effective.
The implications extend beyond cancer drugs. Biosimilars have the potential to significantly reduce the costs of modern health care for people with all kinds of illness, common and rare. And so this decision should be of interest, and concern, to everyone.
I'll close with this filgrastim story: In considering today’s news, I was reminded of a few of my patients who years ago tried to drop off their unused Neupogen vials in the clinic where I worked. Neupogen came in 10-packs, and some – with breast cancer, lymphoma and other conditions – happily wanted to get rid of their last drugs, or what would expire. Although our policy was not to allow patients to “gift” unused cancer drugs one to another, it was clear to the patients how precious these agents were, and are.
* error and correction: 1/7/15, 9 PM: This afternoon the FDA's ODAC unanimously recommended approval of Sandoz's biosimilar version of the drug filgrastim, as indicated. But the FDA has not yet formally approved of the drug, as was suggested in the post's original title (now corrected).
