Jazz’s Defitelio approved in US for rare liver disease

The US regulator has approved Jazz Pharmaceuticals’ Defitelio (defibrotide) for a rare, life-threatening liver condition that can occur after stem cell treatment for blood and autoimmune disorders.

Jazz said that Defitelio is a potentially curative treatment for hepatic veno-occlusive disease (VOD) with renal or pulmonary dysfunction, following haemotopoietic stem-cell transplantation (HSCT).

As there are no other treatments for the condition, the FDA granted a priority review for Defitelio when Jazz filed it in September, granting a six month review instead of the standard ten.

Already approved in Europe in 2013, the drug generated sales of $70.7 million in 2015 for the Irish biotech.

When Jazz bought the drug’s original developer, Gentium, at the end of 2013 for $1 billion, analysts from valuationlab predicted peak sales of around 270 million euros, including US sales. If Jazz can gain a prevention indication, this could add another 190 million euros.

The FDA approval of Defitelio is supported by efficacy data from three clinical studies in patients with hepatic VOD with renal or pulmonary dysfunction following HSCT who were treated with Defitelio at the recommended 6.25 mg/kg every six hours.

The most common adverse reactions with Defitelio treatment were low blood pressure, diarrhoea, vomiting, nausea and nose bleeds.

The most common serious adverse reactions were hypotension (11%) and pulmonary alveolar haemorrhage (7%).

HSCT is an aggressive, potentially curative procedure to treat patients with malignant and non-cancerous haematologic disorders such as leukaemia, lymphoma and aplastic anaemia, and congenital immunodeficiency and autoimmune disorders.

VOD is a rare and life-threatening complication, affecting 9-14% of HSCT patients, affecting the sinusoidal endothelial cells of the liver.

Typically occurring within the first 21 days following HSCT, VOD becomes life-threatening in 30-50% of cases. In VOD with multi-organ dysfunction, mortality rate is 84%.

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