Jazz Pharmaceuticals Is Focused on Label Expansion of Defitelio

Jazz Pharmaceuticals’ Defitelio has managed to penetrate around 80% of the pediatric hepatic veno-occlusive disease (or VOD) market, while its penetration of the adult market is close to 60%. In addition to VOD treatment, Jazz Pharmaceuticals also plans to evaluate the efficacy of Defitelio for the treatment and prevention of diseases due to endothelial cell damage such as graft versus host disease, capillary leak syndrome, engraftment syndrome, diffuse alveolar hemorrhage, transplant associated microangiopathy, and idiopathic pneumonia syndrome. All of these conditions can occur as complications of allogenic or autologous hematopoietic stem cell transplant (or HSCT) and can subsequently lead to multi-organ dysfunction.

Defitelio could help Jazz Pharmaceuticals become a prominent player not only in the treatment but also in the prevention of post-HSCT complications. This segment is currently dominated by peers such as AbbVie (ABBV), AstraZeneca (AZN), and Celgene (CELG).

The above diagram shows the opportunity in the graft versus host disease (or GvHD) markets in the US and Europe.

Starting in 4Q17, Jazz Pharmaceuticals also plans to study the efficacy and safety of Defitelio as a preventative therapy in GvHD indications in a phase two study. The company had scheduled interactions with the FDA and European Medicines Agency (or EMA) in 1H17.

Jazz Pharmaceuticals is currently involved in enrolling patients in a phase three trial to evaluate the potential of Defitelio in preventing VOD in high-risk patients. This study is scheduled to be conducted at 100 sites around the world.

If Jazz Pharmaceuticals succeeds in establishing the drug as a prophylactic therapy in VOD indications, it could push up the company’s share prices as well as those of the Pharmaceutical ETF (PPH). Jazz Pharmaceuticals makes up about 2.8% of PPH’s total portfolio holdings.

In the next article, we’ll explore the market opportunity for Vyxeos in greater detail.