Acute porphyria is designated as an orphan disease by the FDA. Panhematin is the only FDA-approved therapy for acute porphyria, which makes the therapy vital for the treatment of the disorder that if left untreated may lead to long-term or permanent paralysis, coma or even death. Approximately 5,000 people nationwide in the US have the genetic predisposition for the condition and only 20 percent of this population actually develops symptoms.
“Acute porphyria is a very rare condition and few companies would be willing to invest in the area,” said Joseph Bloome, professor of medicine at the University of Alabama Liver Center.
Many pharmaceutical companies choose to form strategic manufacturing relationships because of the complexities associated with the sterile manufacturing and specialized expertise required in manufacturing treatments for rare diseases.
Ovation commented that the new manufacturing facility at Cardinal Health would enable them to provide a constant supply of the drug to patients with acute porphyria by simplifying the manufacturing and packaging process.