The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat from BioCryst Pharmaceuticals, Inc. a positive scientific opinion through the Early Access to Medicines Scheme (EAMS).
Under the EAMS, HAE patients in the UK 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 (EC).
Medicines included in the 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 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.
“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, CEO of BioCryst.
The European Medicines Agency (EMA) is reviewing the marketing authorization application (MAA) for berotralstat under the centralized procedure. 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.