- The Food and Drug Administration on Thursday approved the first medicine for a rare form of anemia, marking an important milestone for its developer, Agios Pharmaceuticals, which recently shifted its research efforts to focus solely on blood diseases and uncommon genetic disorders.
- Agios' medicine, known as mitapivat, is now cleared for use in adults with pyruvate kinase deficiency, a disease characterized by chronically low levels of red blood cells. The FDA based its decision on two late-stage clinical trials that, respectively, found treatment with mitapivat appeared to increase red blood cell counts and decrease the need for blood transfusions.
- Agios is selling its drug under the brand name Pyrukynd, and has set an initial annual price of just under $335,000. The Cambridge, Massachusetts-based company also has plans to get Pyrukynd approved in two other rare blood disorders: sickle cell anemia and thalassemia.
The FDA's latest approval, while significant for patients, is also a crucial victory for a newly redesigned Agios.
Founded in 2007, Agios became best known for its work in cancer research, ultimately developing two marketed drugs for leukemia. But in late 2020, the company announced it would be selling off its cancer treatment business to the French pharmaceutical firm Servier in a deal worth at least $1.8 billion. Going forward, Agios said it would concentrate on rare blood conditions.
At the top a five-year strategic plan devised by Agios was approval of Pyrukynd across three diseases. The nod in PK deficiency is a first step toward that goal, and provides the company with a commercial product to anchor the business.
"Each of us at Agios is dedicated to making a difference for people with PK deficiency, as well as to expanding the reach of Pyrukynd and our clinical and research programs to many more patients with genetically defined diseases around the world," said Jackie Fouse, CEO of Agios, in a statement Thursday.
At nearly $335,000, Pyrukynd follows a trend in rare disease drugmaking of high price tags. While costly, such treatments are still typically covered by insurance companies due to a lack of alternative options.
Agios has been "actively engaging with payers," but coverage may not happen right away, according to analysts at the investment bank Raymond James. They expect the company to secure coverage from commercial payers within the first year of Pyrukynd's launch, whereas a policy with the Centers for Medicare and Medicaid services would take "slightly longer."
Raymond James analyst Danielle Brill highlighted how Agios has so far identified just about 430 patients in the U.S. who could be candidates for its drug. "Considering these dynamics, we continue to expect a very slow launch rollout," Brill wrote in a note to clients.
For Agios, a successful rollout will be helpful in offsetting costs elsewhere in the business. The company has come under pressure in the past for how much it spends on drug discovery and development. Over the first nine months of last year, for example, it recorded $184 million in research and development expenses, while reporting a net loss from continuing operations of $258 million.
Correction: A previous version of this article misspelled the name of Pyrukynd.