The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a positive scientific opinion to the medicine berotralstat for hereditary angioedema patients through the Early Access to Medicines Scheme (EAMS).
BioCryst Pharmaceuticals, Inc., which has several ongoing development programmes including ORLADEYOTM (berotralstat), made the announcement that under the EAMS, patients in the UK aged 12 years and older who live with the rare genetic illness, hereditary angioedema (HAE), will be able to gain access to the oral, once-daily medicine berotralstat.
The medicine can be taken for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorisation by the European Commission (EC).
Early access for patients
HAE is a serious, and potentially life-threatening, rare genetic illness characterised by periodic episodes of acute swelling of the skin, pharynx, larynx, gastrointestinal tract, genitals, and extremities.
The EAMS scheme allows early access to medicines that have a high unmet need, and which are intended to treat, diagnose, or prevent seriously debilitating or life-threatening conditions where there are currently no adequate treatment options available, and which are likely to offer significant advantage over methods currently used in the UK. 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.
Dr Sorena Kiani, Consultant Immunologist at Royal London Hospital, London, said: “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.”
“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 currently reviewing the marketing authorisation application (MAA) for berotralstat and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected approximately 12 months from MAA validation, which the company announced on 30 March, 2020.