Drug for Rare Genetic Disease Gets FDA Approval

Angiodema Drug Approved By FDA
HealthDay News


THURSDAY, July 17, 2014 (HealthDay News) -- Ruconest has been approved by the U.S. Food and Drug Administration to treat hereditary angioedema, a genetic disease that leads to sudden and potentially fatal swelling of the hands, feet, limbs, face, intestinal tract or airways.

The disease, affecting as many as 10,000 people in the United States, is caused by the body's inability to produce enough of a plasma protein called C1-esterase inhibitor. The remedy is produced from the milk of genetically-modified rabbits, the FDA said Thursday in a news release.

Ruconest was evaluated in a clinical study of 44 adults and adolescents with hereditary angioedema. The most common side effects recorded were headache, nausea and diarrhea.

Ruconest is manufactured by the Netherlands-based Pharming Group NV, and will be distributed by a subsidiary of Salix Pharmaceuticals, based in Raleigh, NC.

Copyright © 2014 HealthDay. All rights reserved.

Published: July 2014

ADVERTISEMENT

Suffering From Chronic Pain as a Black Woman

Bias can lead to disparities in diagnosis and treatment of Black women with chronic pain

Chronic Care Issues

Your Child’s Vaccines: What You Need to Know About Catching Up During the COVID-19 Pandemic

Vaccination rates dropped by as much as 60% in some parts of the country due to COVID-19. It's time to get back on track

Prevention & Screenings

Corralling the Facts on Herd Immunity

The effectiveness of herd immunity is a hotly debated topic. Time to separate fact from fiction

Prevention & Screenings