The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended the approval of Takhzyro (lanadelumab) for the routine prevention of hereditary angioedema (HAE) attacks in children starting at age 2. Based on a positive opinion from the CHMP, the European…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

The European Medicines Agency has validated BioCryst Pharmaceuticals‘ application requesting the approval of berotralstat as a once-daily oral treatment to prevent swelling attacks in people with hereditary angioedema (HAE). The Committee for Medicinal Products for Human Use, an advisory committee for the European Union’s regulatory agency, will…

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended Ruconest (conestat alfa) for approval as treatment for acute swelling episodes in children with hereditary angioedema (HAE). In the European Union, Pharming‘s Ruconest is approved for adults…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) — launched…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

The European Medicines Agency (EMA) has approved a type II variation for a new facility to produce Ruconest (conestat alfa), Pharming’s lead therapy for acute attacks in hereditary angioedema (HAE). The new facility is expected to be fully operational by 2021 and to increase the production…

The European Medicines Agency (EMA) will begin an accelerated review of Shire’s lanadelumab, a treatment candidate to prevent hereditary angioedema (HAE) swelling attacks, the company announced. This follows the EMA’s acceptance of Shire’s marketing authorization application (MAA) for the potential therapy, which reduces the number of days…