Jazz pushing potential $1B rare disease drug through rolling NDA
- Defibrotide, developed by Jazz Pharmaceuticals, is intended to treat patients who develop severe veno-occlusive disease (VOD), which is 80% fatal, when undergoing stem cell transplant procedures.
- VOD is a complication of hematopoietic stem cell transplantation (HSCT), which is used for curative purposes in patients with various types of hematologic and solid tumor cancers, as well as some non-cancerous conditions.
- Defibrotide was granted approval for use in exceptional circumstances in HSCT patients by the EU's EMA in October 2013.
Because severe VOD has a 80% fatality rate and is associated with multi-organ failure, the disease truly represents an unmet medical need—a broad area where Jazz Pharmaceuticals focuses its attention.
In addition to being fast-tracked, defibrotide also has an orphan drug designation. By submitting defibrotide for rolling review, Jazz will be able to submit discreet parts of its NDA and get feedback from the FDA one section at a time. This approach can be helpful when a company submits a drug that requires special monitoring and oversight.