Ruconest approved for rare genetic disease

(HealthDay)—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.

More information: The FDA has more about this approval.

add to favorites email to friend print save as pdf

Related Stories

Sivextro approved for adult skin infections

Jun 23, 2014

(HealthDay)—The antibiotic Sivextro (tedizolid phosphate) has been approved by the U.S. Food and Drug Administration to treat adults with skin infections.

Research Lends Hope for Patients With Rare Swelling Disease

Aug 10, 2010

(PhysOrg.com) -- The results of two recent clinical trials offer promising treatments for sufferers of the rare genetic disease hereditary angiodema, says Jonathan Bernstein, MD, an internationally recognized allergy specialist ...

Impavido approved to treat tropical parasitic disease

Mar 20, 2014

(HealthDay)—Impavido (miltefosine) has been approved by the U.S. Food and Drug Administration to treat adults with leishmaniasis, a tropical disease caused by a parasite that's transmitted by the bites of sand flies.

Recommended for you

Drug research and development more efficient than expected

Feb 27, 2015

Drug R&D costs have increased substantially in recent decades, while the number of new drugs has remained fairly constant, leading to concerns about the sustainability of drug R&D and question about the factors that could ...

Use new meningitis vaccines only for outbreaks

Feb 26, 2015

(AP)—A U.S. panel on Thursday recommended that two new meningitis vaccines only be used for rare outbreaks, resisting tearful pleas to give it routinely to teens and college students.

New antibiotic avycaz approved

Feb 26, 2015

(HealthDay)—The combination antibiotic Avycaz (ceftazidime-avibactam) has been approved by the U.S. Food and Drug Administration to treat adults with complicated infections of the intra-abdominal area or urinary tract, ...

Tagging drugs to fight counterfeit medicines

Feb 25, 2015

The U.S. and other countries are enacting rules to clamp down on the sales of fake pharmaceuticals, which pose a public health threat. But figuring out a system to track and authenticate legitimate drugs still faces significant ...

User comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.