What Does Jazz Pharma's Latest FDA Approval Mean?

The US FDA granted marketing approval for Defitelio® [defibrotide sodium] for the treatment of adult and pediatric patients with hepatic VOD, also known as sinusoidal obstruction syndrome [SOS], with renal or pulmonary dysfunction following HSCT. Jazz Pharmaceuticals plc - Ordinary Shares (NASDAQ: JAZZ) will ship to distribution within a week.

Northland Capital Markets’ David Buck maintained an Outperform rating for the company, with a price target of $175, saying that Defitelio had received its first FDA approval for severe liver disease and its launch was “imminent.”

Analyst David Buck expects Defitelio to be a key growth driver and to reach 8 percent of the company’s 2016 revenue estimate of $122M, versus 5.3 percent in 2015. He wrote, “With double-digit volume growth, 2016 EU sales should be low-$80M so guidance of $100-$125M (Northland- $122M) appears an achievable target.”

Severe VOD has a fatality rate of about 84 percent at Day +100, after stem-cell transplant. “Based on Defitelio's FDA-approved label, expected survival rates at Day +100 for non-Defitelio-treated patients are just 21% to 31%,” the analyst mentioned. He added that for the US launch, significant efforts would probably be made to educate physicians on diagnosis and importance of early intervention.