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Profitable Pharming Expands Ruconest Potential And Resurrects Pompe Pipeline

 
• By Jo Shorthouse

Dutch rare disease company Pharming talked to Scrip about turning a profit for the first time in the company’s history, finally restocking the pipeline in potentially blockbuster indications, and turning its attention back to Pompe disease after 20 years in the cold.

Rabbit
Pharming's Ruconest is based on recombinant protein produced in the milk of transgenic rabbits • Source: Shutterstock

The last two years have been transformational for the Leiden-based specialty company Pharming Group NV. In 2016, it had effectively lost control of the commercialization of its intravenous hereditary angioedema (HAE) product, Ruconest, a recombinant human C1 esterase inhibitor (rhC1INH), produced in rabbits, through a series of licensing deals on both sides of the Atlantic, but now its fortunes have turned.

The drug was approved by the US FDA and in Europe in 2014, but Pharming did not market the drug...

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