RARE Daily

BioCryst Drops Development of FOP Program

November 2, 2022

Rare Daily Staff

BioCryst Pharmaceuticals said it is stopping its oral experimental therapeutic BCX9250 for the treatment of patients with the ultra-rare disease fibrodysplasia ossificans progressiva because it believes it is behind other companies’ development programs.

The company made its announcement in its third quarter 2022 financial report. “Considering the expectation that patients will be well-served by these other products, and the approximately $100 million in additional investment that would be required to advance BCX9250 to approval, the company is stopping the BCX9250 program and redirecting this investment to the other opportunities it has to serve patients with complement-mediated diseases,” BioCryst said in the statement.

Fibrodysplasia ossificans progressive (FOP) is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as heterotopic ossification, which can be preceded by painful soft tissue swelling or “flare-ups.” Flare-up episodes are common and are a substantial contributor to the formation of new bone, however bone can also form in the absence of a flare-up. Once formed, it is irreversible and leads to loss of mobility and shortened life expectancy. It is an ultra-rare genetic disorder.

BioCryst said it will focus on advancing its complement programs and expanding global approvals and sales of Orladeyo, its oral treatment to prevent attacks in hereditary angioedema patients, which received U.S. marketing approval at the end of 2020.

“With Orladeyo on a trajectory to more than double sales in its second year of launch after a very strong first year, we continue to demonstrate that what we are offering HAE patients is unique and fills their need for a therapy that offers both low burden of disease and low burden of treatment. We believe that our ability to successfully pursue challenging targets like plasma kallikrein is just the start, and we expect we will bring rare disease patients many more oral therapies to offer these patients the unique treatment options they are waiting for,” said Jon Stonehouse, president and CEO of BioCryst.

Photo: Jon Stonehouse, president and CEO of BioCryst

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