1. Scrip
  2. >>Rare Diseases

All Eyes On US Approval After Ipsen’s FOP Candidate Rejected By EU

Second Time Lucky?

 
• By Ayisha Sharma

The French firm’s palovarotene for fibrodysplasia ossificans progressiva received a thumbs down from the European Commission, but a positive advisory committee outcome on the other side of the pond bodes well for its second attempt at US approval.  

red cross graphic in hand
The EU Has Rejected The Drug After A CHMP Negative Opinion • Source: Shutterstock
Key Takeaways

  • The rejection follows on from a negative CHMP opinion issued in January.

After a controversial clinical development path, Ipsen SA’s palovarotene has been rejected in the EU for the treatment of the ultra-rare bone disorder fibrodysplasia ossificans progressiva (FOP), but an upcoming decision in the US could yield a better outcome given recent events.

FOP causes permanent new bone formation in the soft and connective tissues, a process known as heterotopic ossification (HO). The...

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

Capricor Gets Complete Response For DMD Cell Therapy

 
• By Joseph Haas

Seven weeks before its action date, Capricor got an FDA complete response for cell therapy deramiocel in DMD-related cardiomyopathy. It has a Phase III study nearing readout, though.

In Brief: Soleno To Raise $200m To Boost Vykat XR Launch

 
• By Manas Mishra

Soleno Therapeutics will raise about $200m gross proceeds from an offering of 2.35m of its common stock to fund the commercialization of Vykat XR, which became the first drug for Prader-Willi syndrome to gain US approval on March 26.

Ultragenyx and Mereo Still Confident Despite Setrusumab Slip Up

 
• By Kevin Grogan

The osteogenesis imperfecta drug stumbled in a second interim analysis.

KalVista’s Delayed FDA Approval In HAE Comes Through

 
• By Joseph Haas

KalVista’s Ekterly obtains US approval as the first oral, on-demand therapy for hereditary angioedema. It will compete with established injectables.

More from Scrip

Hopes Rise For Sickle Cell Gene Therapy Access As 33 States Join CMS Program

 
• By Alaric DeArment

CMS said that 33 US states plus the District of Columbia and Puerto Rico had joined a program whereby the agency negotiates outcomes-based contracts on their behalf.

Deal Watch: Debiopharm Takes On Precision Oncology Drug From Repare Collab

 
• By Joseph Haas, Manas Mishra, and Sushmita Panda

Plus deals involving Novartis/Sironax, Neuraxpharm/Dizlin, Recursion/RallyBio, MaaT/Clinigen, Future Pak/Theratechnologies, BerGenBio/Oncoinvent, Xoma/Turnstone and more.

J&J Delivers In Q2 Despite Stelara Drop, Raising Guidance

 
• By Jessica Merrill

Stelara revenues declined 42.7% in the quarter due to biosimilars, but innovative medicines grew 4.9%, surpassing $15bn for the quarter. Although pharma tariffs could be coming, the firm said the impact continues to be hard to predict.