BioCryst wins approval for HAE pill and charges half-a-million dollars, but will anyone take it?
Six years after the FDA OK’d their flu antiviral peramivir, BioCryst has notched another approval.
The North Carolina-based biotech announced Thursday that the FDA had approved berotralstat, a daily pill meant to prevent painful episodes in patients with the rare disease hereditary angiodema (HAE). The molecule, which Biocryst has been studying in the clinic since 2013, will be sold under the name Orladeyo.
Despite inferior efficacy, BioCryst will charge $485,000 for Orladeyo, roughly in line with other treatments on the market. The price tag surprised RBC’s Brian Abrahams, who had projected a $300,000 tag.
The question is how many patients will actually buy it. The molecule met its primary endpoint in a 96-patient Phase III trial last year, successfully reducing the number of painful incidences by 44% compared with placebo. But the stock fell precipitously after BioCryst announced the results, as investors compared that 44% with the reductions shown by other drugs on the market.
BioCryst sought to carve out a niche as one of the only oral options for patients, an alternative to the injectables that have dominated the market. But the drug looked like it might be inferior enough that few patients would switch and risk an increase in HAE attacks, which can be fatal.
Takeda’s Takhzyro, for example, had shown an 87% reduction in painful events, or roughly double.
Abrahams laid out the situation plainly in a note late Thursday night. He predicted around 12% of mild HAE, 9% in moderate, and 7% of severe patients would take the drug, leading to sales around $170 million per year. After BioCryst announced the price, he said sales may be as high as $280 million.
“This marks an important milestone for BCRX, though it is not surprising and was mostly expected given the straightforward, statistically significant data, lack of an AdComm, and high confidence publicly expressed by management as recently as yesterday,” he said. “The real test will be commercial uptake and what proportion of HAE patients will be drawn by Orladeyo’s differentiated oral administration rather than the currently available every-2week SC that has twice the labeled efficacy.”
BioCryst $BCRX shares immediately spiked post-market Thursday but have since slid to $4.77, or 35 cents below yesterday’s $5.12 close.
With BioCryst’s pill underwhelming, other companies, such as the Swiss startup Pharvaris, are trying to come up with their own oral options. BioCryst, meanwhile, is trying to see how effectively their pill can not only prevent attacks but stop them when they occur.