Can This Rare Disease Drug Treat COVID-19?

According to a story from Barron’s, the biotechnology company Alexion Pharmaceuticals has recently announced plans to test its drug Ultomiris as a potential treatment for COVID-19. As the scientific community urgently seeks the development of more effective treatments (and eventually a vaccine) for COVID-19, Alexion is just one of many companies attempting to repurpose one of its own drugs in an attempt to treat the virus.

Can Ultomiris Beat the Virus?

Ultomiris is not an experimental drug and has already received approval from the US Food and Drug Administration for the treatment of two rare illnesses: paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. An extreme inflammation response has been found to be a major mechanism that can potentially cause death in COVID-19 patients with severe disease who have progressed to pneumonia. Ultomiris is classified as an inhibitor of complement component 5 (C5), a protein linked to inflammation.

Trial Begins Soon

Alexion claims that preclinical data indicates that C5 inhibitors could work to bring down inflammation in an animal model of viral pneumonia, as well as anecdotal findings from patients that had received Ultomiris under compassionate use. The company is moving forward with a phase 3 clinical study that will include 270 patients and is geared to start in May. The study will investigate overall survival, duration of hospital stays, and duration of ventilator use. Alexion also announced that it will offer its drug Soliris, another C5 inhibitor, to severe COVID-19 patients in France and the US under expanded access protocols.

The company joints the ranks of Eli Lilly, Roche, Sanofi, and Regeneron Pharmaceuticals in testing their own anti-inflammatory drugs as possible treatments for COVID-19. While only time will tell if any of these therapies will have any sort of meaningful impact on the virus, the urgent need for better treatments means that there will probably be further drug companies that jump on the bandwagon in the coming months.

Ultomiris and Soliris are extremely expensive drugs, with a year’s supply running about $500,000. When asked about how pricing would be affected if the trial is a success, a spokesperson for the company replied:

“We are working to prepare for this and other potential scenarios that may arise so that we are ready to support access to, and anticipated demand for, our medicines in the future.”


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