Orladeyo Set to Launch by Year’s End if Approved, BioCryst Says

BioCryst Pharmaceuticals is prepared to launch Orladeyo (berotralstat), an oral treatment for hereditary angioedema (HAE), by the end of the year if the medication is approved, the company has announced.

“We are currently in an exciting transformation from a company primarily focused on R&D [research and development] to one that is about to launch its first oral drug to patients with HAE (Orladeyo) this year,” Jon Stonehouse, president and CEO of BioCryst, said in a press release.

Orladeyo is an investigational HAE treatment that works by lowering the levels of bradykinin, an inflammatory molecule that promotes swelling, the overproduction of which characterizes HAE.

Data from the clinical trials APeX-2 (NCT03485911) and APeX-S (NCT03472040, still recruiting) have demonstrated that the medication can significantly reduce the frequency of HAE attacks and improve quality of life. The treatment was generally well-tolerated, with the most common side effect being the common cold.

“Our ongoing dialogue and research with HAE patients and physicians continue to reinforce their strong demand for an oral, once-daily medicine that is safe and provides the significant and sustained attack reduction we are seeing with Orladeyo in our clinical program,” said Charlie Gayer, chief commercial officer of BioCryst.

“Nearly half of patients in our APeX-2 and APeX-S trials have prior experience with injectable or infused therapies and most have chosen to remain on Orladeyo,” Gayer added.

BioCryst has submitted applications requesting the approval of Orladeyo to multiple regulatory bodies around the world, including the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and Japan’s Pharmaceutical and Medical Devices Agency (PMDA).

Decisions from the FDA and PMDA are expected by December. An opinion from the EMA’s Committee for Medicinal Products for Human Use is expected by March 2021.

“We expect to end the year with Orladeyo approved in the U.S. and Japan,” Stonehouse said.

The company stated that it is well-positioned in terms of product supply and inventory to meet the anticipated demand for Orladeyo, if the medication is approved.

In addition, BioCryst recently announced the establishment of an expanded access program in the U.S., which allows licensed physicians to request Orladeyo for people with HAE who do not have access to it through a clinical trial.