According to a story from biopharmadive.com, the pharmaceutical company Roche is reporting a total of five deaths of patients with hemophilia A that have taken the company’s product Hemlibra. While suspicious, it has been determined in these cases that the drug is not necessarily the cause of the incidents.
Hemophilia is a genetic disorder in which the body’s ability to form blood clots is affected. Patients may not be able to form clots or may take much longer to do so. Forming blood clots is an essential part of the wound healing process, and people with hemophilia tend to bleed for a longer period of time after being injured compared to unaffected people. In addition, hemophilia patients are at risk of internal bleeding in the joints and brain, which can cause long term damage and severe complications if left unchecked. Hemophilia is caused by the body not producing sufficient quantities of clotting factor, or not producing them at all. Typically, treatment involves supplementing the body’s supply of the missing factor. To learn more about hemophilia, click here.
Hemlibra is developed for patients with severe hemophilia A, in which clotting factor VIII is deficient. While normally supplementing the factor is sufficient, some patients have developed antibodies that prevent the introduced factor VIII from working. Hemlibra is an alternative that is meant to help those patients. This is not the first time that there have been safety issues with Hemlibra, and the medication carries a black box warning for severe side effects.
Of the five deaths reported, the company says that the investigators determined that the drug was not the cause.* Considering the severity of the disease in patients who take the drug, it is not entirely uncommon for deaths to occur despite treatment. However, this doesn’t completely get Hemlibra off the hook.
The deaths come at a difficult time for Roche, who ultimately hopes to make Hemlibra available to a larger field of patients. So far, the company remains confident in the drug’s safety profile and there haven’t been any changes to the benefit/risk profile approved by the FDA. At the end of the day, while the drug can still be considered safe, the hemophilia community would be wise to stay vigilant for any additional reports about the safety profile of the Hemlibra.
*Correction 4/11/18–This article originally stated that only three of the five cases had been attributed to other causes. New information has determined that the drug was not implicated as the cause in all five deaths.