RESEARCH TRIANGLE PARK – The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Durham-based BioCryst Pharmaceuticals’ berotralstat a positive scientific opinion through the Early Access to Medicines Scheme.

Berotralstat is an oral, once-daily medication for hereditary angioedema (HAE), a serious and potentially life-threatening, rare genetic illness characterized by periodic episodes of acute swelling in various parts of the body including skin, throat, gastrointestinal tract and extremities.

With the regulatory agency’s positive opinion, hereditary angioedema patients in the U.K. aged 12 years and older can gain access to berotralstat for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the European Commission.

“HAE patients around the world are waiting for an oral, once-daily therapy to prevent attacks and reduce their burden of therapy,” said Jon Stonehouse, chief executive officer of BioCryst. “With this decision by the MHRA, the wait for many HAE patients in the U.K. can end sooner.”

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Medicines included in the United Kingdom’s Early Access to Medicines Scheme (EAMS) are those that have a high unmet need, are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where there are no adequate treatment options, and are likely to offer significant advantage over methods currently used in the country. Under the scheme, the MHRA provides a scientific opinion on the benefit-risk balance of the medicine, based on the data available when the EAMS submission was made.

“There are many patients in the U.K. that don’t have a realistic option for effective HAE prophylaxis,” said Dr. Sorena Kiani, consultant immunologist at Royal London Hospital, London. “The addition of berotralstat through the EAMS will bring a much needed option for HAE patients suffering with this debilitating disease.”

The European Medicines Agency is reviewing the marketing authorization application for berotralstat under the centralized procedure. An opinion from the Committee for Medicinal Products for Human Use is expected approximately 12 months from the marketing authorization application validation, which the company announced on March 30, 2020.

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