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FDA Grants Breakthrough Therapy Designation to Actemra for Giant Cell Arteritis
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The FDA has granted a breakthrough therapy designation to Actemra for the treatment of giant cell arteritis, for which no therapies have been approved in more than 50 years, according to a Genentech press release.
“The FDA breakthrough therapy designation for [giant cell arteritis] GCA underscores our continued commitment to explore Actemra in autoimmune diseases with significant unmet need,” Sandra Horning, MD, chief medical officer and head of global product development at Genentech, said in the release. “We are looking forward to working with the FDA in the hope of making Actemra available to people with GCA ... ”
The breakthrough therapy designation is designed to speed the development for treatments of serious diseases. Current treatment for GCA has been limited to high-dose steroids, which can prevent vision loss but fail to prevent long-term remission of the disease, the release noted. Patients with GCA are typically seen by rheumatologists, neurologists and ophthalmologists due to the complexity of the disease.
In June, Genentech announced the results of the phase 3 GiACTA study that assessed Actemra (tocilizumab, Genentech) — which is currently approved as a second-line treatment for rheumatoid arthritis — in patients with GCA and compared it to currently used steroid treatment.
“Steroids are an essential sight-saving tool, but can be as harmful long term at moderate to high doses systemically as they are helpful to saving sight,” Alan L. Wagner, MD, FACS, retina specialist and founder of Wagner Macula & Retina Center, said in the release. “The phase 3 tocilizumab study is a large step forward for our patients with this chronic autoimmune disorder.”
The results showed tocilizumab, when combined with 6 months of glucocorticoids, more effectively sustained remission after 1 year compared with glucocorticoid-only regimens.
“The results of the GiACTA study mark a huge step forward for patients with GCA — in more than 50 years, no medication other than glucocorticoids has ever been shown in a convincing way to be effective,” John H. Stone, MD, MPH, lead investigator of GiACTA and professor of medicine at Harvard Medical School, said in the release. “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.”
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Leonard H. Calabrese, DO
On Oct. 5, the FDA granted breakthrough status to tocilizumab (Actemra, Genentech) for the treatment of giant cell arteritis. To be granted breakthrough status, a drug must demonstrate clear efficacy and assume an important place in the therapy of a given disease. To quote the FDA, a breakthrough drug must fit the following description – “preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”
The phase 3 GIACTA trial did just this by achieving both its primary and secondary clinical endpoints and demonstrating that adjunctive tocilizumab, when combined with glucocorticoids for 6 months, was more effective in achieving sustained remission vs. standard glucocorticoid therapy for either 6 months or 12 months. Toxicity was acceptable thus supporting a role for IL-6 inhibition as a “go-to” for steroid refractory cases and maybe more.
Many questions remain, however. Among the remaining questions in my mind are:
- The study was done with high-dose tocilizumab (8 mg/kg). What may be the role of 4 mg/kg in patients with comorbidities or as a dose reduction?
- While toxicity was acceptable in the small trial (less than 300 patients), will this hold up in an elderly population with a lot of glucocorticoid exposure being a dominant risk factor for serious infections, opportunistic infections and gastrointestinal perforations?
- Will adjunctive therapy lower vascular complication rates in a larger population?
- Will it demonstrate equal efficacy in polymyalgia rheumatica as expected?
- Will the newer agents in the IL-6 arena that are in late-stage development also be effective as several trials of these agents are also underway in these disorders?
- How will payers approach the incorporation of IL-6 inhibition in the treatment pathways when approved?
Yes, it is an exciting time with a second biologic agent now approved for patients with vasculitis, which is good news for patient care. Let’s keep up with the continued research and publications in this fast moving area.
I just noted on my cool American College of Rheumatology (ACR) app that abstract #911, “Efficacy and safety of tocilizumab in patients with giant cell arteritis” is the second presentation of the first day’s plenary session. I will be there. Follow me on Twitter throughout the ACR Annual Meeting (#ACR16) at @LCalabreseDO.
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