News

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…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD), said it will still take place at the…

Preventive treatment with berotralstat (BCX7353) significantly lowered the number of swelling attacks — including those needing treatment — and the rates of standard on-demand therapy use in people with hereditary angioedema (HAE), data from a Phase 3 clinical trial show. Notably, use of on-demand therapy fell to a greater extent…

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…

People with hereditary angioedema (HAE) caused by a specific mutation in the plasminogen gene have several on-demand and preventive treatment options that reduce the severity and frequency of their acute swelling attacks, a study has found. The study, “Treatment of Hereditary Angioedema with the Mutation…

BioCryst Pharmaceuticals presented new results from a Phase 3 clinical trial showing that preventive treatment with oral berotralstat (BCX7353) significantly lowers the frequency and may also lessen the severity of swelling attacks in people with hereditary angioedema (HAE). The findings would have been shared at the…

The U.S. Food and Drug Administration (FDA) has approved a new production facility for Ruconest (conestat alfa), Pharming’s lead therapy for acute attacks in hereditary angioedema (HAE). The Netherlands-based facility also has been approved by the European Medicines Agency (EMA). That means…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

Mutations in the KCNMA1 gene may predispose certain people to angioedema caused by blood pressure medications, a study suggests. The study, “Genome-wide association study of angioedema induced by angiotensin-converting enzyme inhibitor and angiotensin receptor blocker treatment,” was published in The Pharmacogenomics Journal. Angiotensin-converting enzyme inhibitors…