#BCRX "announced that the United Kingdom’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).
Under the EAMS, hereditary angioedema (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).
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/or extremities.
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" (see press release HERE).
See our BCRX AmpCard for Berotralstat and its upcoming catalysts.