BioCryst takes on Takeda, CSL with FDA approval for Orladeyo, the first pill to prevent HAE attacks

BioCryst Pharmaceuticals won FDA approval for oral HAE medicine Orladeyo on Thursday. (BioCryst)

Ready to take on established competition from Takeda and CSL Behring, BioCryst Pharmaceuticals has scored FDA approval for Orladeyo, the first oral option to prevent hereditary angioedema (HAE) attacks in patients 12 and older.

While the med carries a high list price of $485,000 per patient per year, it comes in below the prices for Takeda’s Takhzyro and CSL’s Haegarda, Evercore ISI analyst Liisa Bayko wrote in a note to clients. Both of those medicines are administered through injections, and both generate significant revenues for their respective companies.

While BioCryst's Orladeyo won’t be available until later this month and the analyst doesn’t expect meaningful sales this year, Bayko predicts the drug will grow to $300 million in 2023 U.S. revenues. Patients and doctors have been “eagerly awaiting” an oral treatment option, Bayko wrote, predicting that by the end of 2021 Orladeyo will reach around one-third of the preventive HAE market.

RELATED: BioCryst says oral drug works for HAE attacks

In a phase 3 trial, Orladeyo significantly reduced HAE attacks at 24 weeks; the benefit was sustained through 48 weeks. The medicine reduced attacks from 2.9 per month on average to one per month.

Doctors can begin writing Orladeyo prescriptions immediately, BioCryst says, and the company’s specialty pharmacy partner will begin direct-to-patient shipments by the end of the month.

Meanwhile, BioCryst has partnered with Torii Pharmaceuticals for a rollout in Japan, and an approval could come later this month, Bayko wrote. Regulators in Europe are set to decide on the drug in the first half of 2021.

RELATED: BioCryst starts COVID-19 trial of broad-spectrum antiviral

Aside from its work in HAE, the company is advancing several other drug candidates, including a broad-spectrum antiviral, galidesivir, that’s being investigated as a COVID-19 therapy.