After delay, Kalvista wins FDA OK for drug to treat rare swelling disorder
Dive Brief:
After a delay due to 'resource constraints,' the Food and Drug Administration on Monday approved Kalvista Pharmaceuticals' pill Ekterly to treat swelling attacks in people with the rare disorder hereditary angioedema.
Ekterly is the first oral drug to treat hereditary angioedema, or HAE, attacks, competing with shots like Firazyr from Takeda and Ruconest from Pharming. Analysts have estimated Ekterly, Kalvista's first marketed drug, could bring in $600 million a year in U.S. sales at its peak.
The FDA delayed the decision beyond its June 17 deadline, Kalvista said, because of a 'heavy workload and limited resources.' While Kalvista awaited its decision, the FDA granted approval to another HAE drug, CSL's Andembry, a preventive shot that won't compete directly with Ekterly.
Dive Insight:
Kalvista set a price of $16,720 per dose, which consists of two 600 milligram tablets of Ekterly, known scientifically as sebetralstat. A company spokesperson stated Kalvista 'priced Ekterly competitively with existing therapies, reflecting the innovation it brings to the HAE community,' noting that it's the first new drug in more than a decade.
Under the FDA's approval, people experiencing a HAE attack can take a second dose within three hours if they don't get symptom relief, while people taking Firazyr must wait six, a protocol that could give Ekterly a competitive advantage, Stifel analyst Paul Matteis wrote in a note to clients. Moreover, people using Firazyr and Ruconest must receive training before they can self-inject.
The drug won approval based on a trial of 110 people, which found that people who took Ekterly after an attack achieved symptom relief in a median of two hours following a dose within a 12-hour window in which the drug's effectiveness was measured. Fewer than 50% of people who took a placebo in the trial experienced symptom relief within 12 hours.
More HAE attacks were resolved in people who took Ekterly within 24 hours, at about 50% compared to 27% in those who took placebo.
Kalvista shares rose 24% in early trading, recovering all of the losses following the FDA's delay.
Matteis took an optimistic view of the drug, writing that Ekterly's 'injectable-like efficacy has the potential to take a meaningful share, and potentially grow, the on-demand HAE market, which has remained relatively stable.'
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