Salix Pharmaceuticals ($SLXP) picked up FDA approval for an orphan therapy, giving the company another revenue source as it plots a tax-saving move to Ireland.
The drug, Ruconest, is designed to treat acute swelling in patients with hereditary angioedema, a genetic disease that affects between 6,000 to 10,000 people and results in pain and swelling in the face, lungs and upper airway. The treatment works by blocking the complement system esterase C1, replacing a protein that helps tamp down allergic reactions in healthy patients.
Ruconest is meant to be administered at the onset of hereditary angioedema attacks, returning patients' C1 inhibitor protein levels to normal and relieving dangerous swelling, Salix said. In its pivotal trial, the drug beat out placebo in providing relief from attacks, according to the company.
Now Salix will have to contend with entrenched offerings from Shire ($SHPG) and Dyax ($DYAX), companies that have been selling treatments for hereditary angioedema attacks for years. However, unlike its competitors' Firazyr and Kalbitor, which block a protein responsible for swelling, Salix's treatment addresses the deficiency at the heart of the disease, a difference the company hopes will help it gain market share.
"Ruconest is a much-needed treatment option for patients suffering from acute attacks of hereditary angioedema," CEO Carolyn Logan said in a statement. "Until now, there hasn't been an FDA approved recombinant C1 esterase inhibitor option to treat symptoms of HAE."
Salix acquired the North American rights to Ruconest in its $2.6 billion cash acquisition of Santarus, leaving the Netherlands' Pharming with rest-of-the-world marketing permission.
Meanwhile, the North Carolina company is waiting on the close of a $2.7 billion reverse merger that'll join it with Cosmo Pharmaceuticals ($COPN) and give it an Irish domicile, lowering its effective tax rate through a process called inversion. Salix joins a growing list of U.S. outfits pursuing such deals, including Medtronic ($MDT), AbbVie ($ABBV) and Auxilium ($AUXL).
- read Salix's statement
- here's the FDA release