Drug Derived From Milk of Genetically Modified Rabbits to Treat Rare Swelling Disorder
July 30, 2014
The FDA has approved new drug, Ruconest, to treat people with Hereditary Angioedema.
The new drug, developed in partnership by Salix Pharmaceutical and Pharming, is intended as a protein replacement treatment for people with hereditary angiodema. The disease affects between 6,000 and 10,000 people in the US and leads to rapid swelling of the hands, feet, limbs, face, intestinal tract and airway. Its triggers include stress and infection.
Because of the rarity and low prevalence of the condition, the FDA has classified Ruconest as an orphan drug – a regulatory classification which will give Salix and Pharming 7 years of exclusive patenting rights for the drug. The approval covers the drug’s use in the treatment of acute attacks of hereditary angioedema in adults and adolescents.
The drug works by increasing and topping up levels of a plasma protein called C1-esterase inhibitor. Insufficient levels of the protein are characteristic of hereditary angioedema.
“Until now, there hasn’t been an FDA approved recombinant C1 esterase inhibitor option to treat symptoms of hereditary angioedema,” said Carolyn Logan, CEO of Salix. “The unpredictability of hereditary angioedema can make patients feel uncertain about when their next attack might strike, which is why it is important to have a medicine that can be administered by the patient that resolves an attack.”
Salix and Pharming have developed a way to produce the replacement protein by breeding rabbits that have the gene that produces the human C1 inhibitor protein. The gene is then harvested from the milk of these rabbits. This means that the use of Ruconest is limited to patients that do not have a history of allergy to rabbits. Previously, genetically modified goats have been used to produce milk containing an enzyme to treat Gaucher’s disease.
The drug has proved its safety and efficacy in clinical trials involving 44 patients who regularly experience acute attacks of hereditary angiodema, with sympton relief being provided within 90 minutes compared to 152 minutes for people that were taking the placebo.
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