Updates on berotralstat

A series of updates on berotralstat, also called Orladeyo, which is currently in some countries, were provided by the pharmaceutical company BioCryst. The key updates were:

• The company has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to expand the berotralstat label to children with HAE aged 2 to 11 using an oral granule formulation. The company also expects to submit regulatory filings in 2025 in global territories, including Europe, Japan and Canada. Berotralstat would be the first targeted oral prophylactic therapy for children with HAE.
• HAE patients who describe a strong preference for an oral prophylaxis therapy increased to 70 percent, up from 50 percent in 2023, in the company’s latest market survey of HAE patients.
• The number of patients treated with berotralstat outside the US continued to grow in new and existing markets.

Dr Helen Thackray, Chief Research and Development Officer of BioCryst, said: “As we move closer to delivering an urgently needed oral prophylactic therapy to children with HAE, we are simultaneously progressing two clinical-stage programs in Netherton syndrome and DME towards the first patient data later this year.”

(Source: BioCryst)