Back in 2010, Actemra (tocilizumab) was approved by the FDA to treat rheumatoid arthritis (RA), an inflammatory condition affecting joints. At the time, with over 1.5 million Americans suffering from RA, Actemra‘s release was promising for many doctors and patients.
Actemra is a first-in-class drug, meaning it works in a new and unique way to to treat RA, polyarticular juvenile idiopathic arthritis (PJIA), systemic juvenile idiopathic arthritis (SJIA), and as of this month—giant cell arteritis (GCA).
So, why the concern now? To understand, it’ll help to follow Actemra through its path to approval.
First approved in Japan:
Actemra was approved in Japan in 2008, and at the time, the side effects were considered minor compared to other RA treatments: high blood pressure, increased liver enzymes, and upper respiratory tract infections. At the time of its approval, the lack of side effects excited the RA community, and Actemra was seen as a breakthrough for patients.
More serious side effects than expected:
A 2009 report showed 15 deaths and over 200 severe side effects in a patients taking Actemra. The deaths weren’t definitively caused by Actemra, but the manufacturer was unable to rule out the possibility that the drug was to blame.
There were concerns that this new-found data would cause a potential setback for future sales in Japan and Europe where the medication was already approved, and in the United States where the manufacturer was still seeking FDA approval. However, even with the new link to severe side effects, Actemra continued to be sold in Japan.
How did Actemra get approved in the US?
The information from these initial studies was used to warn doctors about the side effects of using or prescribing this drug. After the reports in Japan, Actemra went through an extensive clinical development program with 5 multi-national Phase III studies in 41 countries including the United States, which involved more than 4,000 patients prior to its FDA approval. (Fewer than 10% of drugs in development make it through Phase III trials in the US.)
The Phase III studies did show some rare but serious side effects, including serious infections that may lead to hospitalization or death, gastrointestinal perforations (a hole in the stomach or intestines), hypersensitivity reactions, and life-threatening allergic reactions.
Actemra was still seen as a breakthrough drug, with a low enough risk to patients vs its benefits, and was approved in the US in 2010.
Why the concern now?
A recent investigation by STAT revealed that Actemra‘s risk to patients may be greater than previously thought. Evidence has suggested that many patients may have been harmed by Actemra—and not from the serious side effects noted in the prescribing information. Patients taking Actemra have experienced other conditions like heart failure, heart attack, stroke, pancreatitis, and lung disease—none of which are mentioned by the manufacturer in the package insert.
There is a black-box warning (the most serious kind of warning attached to prescription drugs) for Actemra, but only for the possibility of the risk of serious infection, as mentioned above. Iodine also has a good overview of the rest of Actemra‘s reported side effects.
According to STAT, since Actemra‘s approval in 2010, the FDA has received over 1,000 reports of people who have died while taking the RA medication. These investigations have led to multiple safety reviews, but at this point, there is not enough evidence to suggest that Actemra is causing these cardiovascular issues.
What is being done?
Even with the reports of serious side effects, neither the manufacturer, Roche, or the FDA have made efforts yet to investigate the potentially deadly side effects, and warn patients and doctors. The hope is that the STAT investigation may spark the interest of the FDA to review Actemra and update the package labeling.
In the meantime, if you or a loved one is currently taking Actemra, be sure to talk to your doctor about your concerns.
Stay tuned—GoodRx will keep you informed!