Biogen Idec Inc, a Massachusetts-based biotech company, is a leader in the treatment of multiple sclerosis, producing four of the 11 FDA-approved disease-modifying drugs for MS including Avonex, Plegridy, Tysabri, and Tecfidera. Dimethyl fumurate (brand name: Tecfidera), an oral pill taken twice daily to treat relapsing forms of MS, including relapsing-remitting MS, was approved in the United States in March 2013, followed by Canada, Australia, the European Union, and Switzerland. More than 100,000 patients worldwide have used Tecfidera to date.
During Biogen’s 3rd quarter earnings conference call on October 22, 2014, company executives confirmed that a patient who had taken its MS pill, Tecfidera, had been diagnosed with progressive multifocal leukoencephalopathy (PML) and recently died of complications of pneumonia. PML is a rare brain infection that can cause severe disability and results in death in approximately 20% of cases in MS patients. PML is an opportunistic infection connected with reactivation of the JC (John Cunningham) virus in patients with a lowered immune system.
Biogen CEO George Scangos said that the patient had taken Tecfidera for more than four years and experienced persistent lymphopenia (abnormally low white blood cell counts) during most of that time as a clinical trial participant. The Tecfidera label carries a warning for lymphopenia which is a much less common side effect of the drug than gastrointestinal disorders or flushing which affected 18-40% of patients during clinical trials. Lymphopenia is a known risk factor for PML in addition to the presence of antibodies to the JC virus in a patient’s blood.
“This patient did have severe lymphopenia for three and a half years, and we believe the length of time on Tecfidera is not the issue. The issue is related to lymphopenia,” Scangos said on the conference call. “Despite this tragic loss, we believe the risk profile of Tecfidera remains unchanged.”
While Biogen stated that the patient’s death was not due to Tecfidera, but to complications of pneumonia, the company has reported the death to the FDA and is working with the agency to determine whether the labeling for Tecfidera needs to be changed as a result. Last year the death of a 59-year-old woman who took Tecfidera for 5½ weeks was reported and determined to be unrelated to the drug by her doctor.
The FDA encourages healthcare professionals and patients to report serious adverse events, product quality problems, product use error or therapeutic inequivalence/failure related to use of any FDA-regulated drug, biologic, medical device, dietary supplement or cosmetic through the MedWatch program ( http://www.fda.gov/Safety/MedWatch/). You can also report suspected counterfeit medical products to FDA through MedWatch.
During the conference call, Biogen reported positive third quarter 2014 results, including revenues of $2.5 billion which represent a 37% increase compared to the third quarter of 2013. Tecfidera revenues were $787 million in Q3 2014 (up from $286 million last year), consisting of $638 million in the US and $149 million outside the US. Avonex and Plegridy revenues were $745 million, while Tysabri sold $501 million. Biogen expects total revenues to reach $9.8 billion by the end of the year.
Biogen’s monoclonal antibody therapy, Tysabri, is famously known to be connected to an increased risk of developing PML. However, Biogen has worked diligently to stratify patient risk of developing PML based on demonstrated exposure to the JC virus, use of immunosuppressant drugs, and length of time on Tysabri, in order to proactively reduce each patient’s risk. As of September 2, 2014, there have been 495 confirmed cases of PML among patients using Tysabri resulting in 111 deaths, said Kate Niazi-Sai, a spokeswoman for Biogen.
While Tecfidera does not seem to be directly related to this first reported case of PML in a patient using the drug, it is important to remain vigilant in reporting side effects and potential adverse events to our healthcare professionals, as well as the FDA, when we suspect conditions may be related.1-4