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FDA approves Actemra for giant cell arteritis
The FDA has approved Actemra for the treatment of adults with giant cell arteritis.
“Rheumatologists will finally have available an option for their patients that is going to allow them to discontinue steroids as rapidly as possible,” Christine Birchwood, PhD, senior medical science director from Genentech, told Healio Rheumatology. “It is the first new therapy these patients will have had in more than 50 years.”
“Today’s FDA decision means people living with giant cell arteritis will, for the first time, have an FDA-approved treatment option for this debilitating disease,” Sandra Horning, MD, chief medical officer and head of Global Product Development, said in a company press release.
According to the release, tocilizumab (Actemra, Roche and Genentech) is the first FDA-approved therapy for the treatment of adults with giant cell arteritis (GCA). To make its decision, the FDA used data from the phase 3 GiACTA study of 251 patients with GCA. The results showed patients treated with tocilizumab plus a 6-month glucocorticoid regimen more effectively sustained remission through 52 weeks (56% for weekly tocilizumab and 53.1% for bi-weekly tocilizumab) compared with patients who took placebo plus with a 26-week steroid taper (14%) and with patients who took placebo plus a 52-week steroid taper (17.6%). The safety findings were consistent with its known profile, which is an increased risk for serious infection.
“The results of the GiACTA study mark a huge step forward for patients with GCA. For more than 65 years, no medication besides glucocorticoids has been proven in a convincing way to be effective,” John H. Stone, MD, MPH, GiACTA’s lead investigator, professor of medicine at Harvard Medical School, director of Clinical Rheumatology and Edward Fox Chair in Medicine at the Massachusetts General Hospital, said. “This rigorous, randomized and carefully blinded study addresses one of the greatest needs of physicians who treat GCA – finding an alternative to prolonged periods of steroid therapy.”
The FDA previously granted breakthrough therapy designation for this application, as well as a priority review.
In the FDA press release, Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, said, “We expedited the development and review of this application because this drug fulfills a critical need for patients with this serious disease who had limited treatment options.”
“The rheumatology community has shown a lot of enthusiasm about these data,” Birchwood told Healio Rheumatology. “I think they are excited to have an alternative treatment for their patients.”
References:
www.gene.com/media/press-releases/14667/2017-05-22/fda-approves-genentechs-actemra-tocilizu
Perspective
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In my May 2017 Editorial in Healio Rheumatology, which was written a few weeks before the recent FDA approval of tocilizumab for GCA, I said it promised to be a “game changer.” I wrote this not knowing the labeling, but recognizing the definitive nature of the GiACTA study and that it was granted breakthrough therapy designation by the FDA allowing it to be fast-tracked through the regulatory pathway. This designation has been rarely awarded and is most commonly used for oncology drugs.
Now that we have the prescribing information for tocilizumab, we see it was globally approved for the treatment of GCA in adults. We see it was approved for subcutaneous administration only, which will greatly influence the reimbursement issues in this Medicare-enriched population. We also see it was approved at the weekly dose of 162 mg “with a tapering dose of glucocorticoids” — the dose of which was based on “clinical considerations.”
Where does this broad labeling leave us who care for patients with GCA? First, I have not written for this drug yet, but I am eager to hear about how easy or hard it has been for providers to get third parties to approve and reimburse for it. Second, will we still get approval for the reduced dose (once per week), which had respectable outcomes as well? Third, will we get approval for monotherapy, ie, tocilizumab? For as we all know, there are patients with glucocorticoid risks for whom this would be appealing. Note this was not studied in the GiACTA trial, but I know of no other agent with such therapeutic potential in this small and vulnerable population. Lastly, in the “glucocorticoid era,” patients have been documented to remain on steroids in high percentages after 2 years to 5 years, particularly in data reported from referral centers. What will this mean for these patients? Will they be on tocilizumab for this long?
Many questions remain with so little data. Please write to me at calabrl@ccf.org or @LCalabreseDO on Twitter to share your experiences and observations. Let’s get back on the long strange trip of GCA.
Reference:
Calabrese LH. Giant cell arteritis: What a long strange trip it has been. Healio Rheumatology. 2017;2(5):8-9.