Profitable Pharming Expands Ruconest Potential And Resurrects Pompe Pipeline

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 in either territory, as Valeant Pharmaceuticals International Inc and

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Rare Diseases

Roivant Staying ‘Patient’ On Deals, Leaving Investors Waiting

 

CEO Matt Gline talked to Scrip in an interview about dealmaking and the road back to commercialization.

InflaRx Stock Sinks As Vilobelimab Fails Phase III Skin Trial

 
• By 

A study of the German biotech's intravenous complement factor C5a antibody for pyoderma gangrenosum was stopped for futility.

Sweden’s Hansa Slims Down Ahead of Key Readouts

 
• By 

New CEO Renée Aguiar-Lucander is marking her mark.

Zydus To Continue Mirabegron Sales, Looks To World-First Dual Shigella-Typhoid Vaccine

 
• By 

Zydus expects strong mirabegron sales in FY26 amid US litigation even as it builds a growth pillar in vaccines with a world-first, Gates Foundation-aided dual shigella-typhoid vaccine under development and others on the WHO prequalified list

More from Scrip

Merck & Co./Daiichi Pull Patritumab Deruxtecan BLA On Disappointing OS

 

Patritumab deruxtecan is the lead antibody drug conjugate in a $22bn deal signed between the two partners, with the decision marking a disappointing setback

Executives On The Move: Innate Pharma And Alloy Therapeutics Get New CEOs

Pascal Soriot, CEO of AstraZeneca, joins Agilent Technologies' board, plus Zealand Pharma acquires chief development officer from UCB Biopharma.

Senti’s Computer-Logic Inspired Cell Therapy Shows Early Promise

 

The biotech believes its logic-gated cell therapies can get round the need for ‘clean targets’ which limit CAR-Ts and ADCs.