MHRA allows HAE patients early access to berotralstat

Patients with hereditary angioedema (HAE) living in the UK will get early access to BioCryst's berotralstat before the drug is officially approved for this indication by the European Commission.

The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat a positive scientific opinion through the Early Access to Medicines Scheme (EAMS), thus allowing patients aged 12 years and older to receive treatment with the drug for the routine prevention of recurrent attacks of HAE.

HAE is a rare, potentially life-threatening, genetic disorder causing periodic episodes of acute swelling of the skin, pharynx, larynx, gastrointestinal tract, genitals and/or extremities.

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

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

The European Medicines Agency (EMA) is reviewing the marketing authorisation application (MAA) for berotralstat under the centralised procedure; an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected by the end of March 2021.