1. Scrip
  2. >>Rare Diseases

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 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
Start Free Trial

Already a subscriber?

More from Rare Diseases

InflaRx Stock Sinks As Vilobelimab Fails Phase III Skin Trial

 
• By Kevin Grogan

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 Kevin Grogan

New CEO Renée Aguiar-Lucander is marking her mark.

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

 
• By Vibha Ravi

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

Lundbeck Lifted By Leap In Migraine Therapy Sales

 
• By Kevin Grogan

Vyepti revenues rise by nearly 70% in the first quarter.

More from Scrip

Lilly To Diversify Pain Pipeline With SiteOne Acquisition

 
• By Joseph Haas

Deal snapshot: With several internal non-opioid pain candidates in Phase II or earlier, Lilly looks to add R&D potential in pain with $1bn buyout of SiteOne.

Angelini Advances Brain Health Ambitions With GRIN Pact

 
• By Kevin Grogan

The Italian drugmaker gets rights to Phase III-ready asset radiprodil.

GlycoEra’s Series B Cash Will Fund Efforts To Take On FcRn Inhibitor Class

 
• By Joseph Haas

GlycoEra unveiled a $130m series B financing, saying the proceeds would enable it to take its first clinical candidate through proof-of-concept in autoimmune diseases.