Real-World Data Boost Santhera's Latest DMD Filing, And The Markets Agree

Despite being knocked back twice, Santhera CEO Thomas Meier is still confident Raxone has a place as a Duchenne muscular dystrophy therapy, and believes new long-term data will strengthen its third application to the EU in the coming months.

Dart
Could new data hit the bullseye? • Source: Shutterstock

Having been turned back twice by the European regulators in its pursuit of a Duchenne muscular dystrophy (DMD) indication for Raxone (idebenone), Santhera Pharmaceuticals AG has published the real-world data it believes will prove the doubters wrong.

Latest results from the SYROS study have shown that long-term treatment with idebenone, marketed for Leber’s hereditary optic neuropathy (LHON) in Europe, consistently reduced the rate of respiratory function

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

Lundbeck Lifted By Leap In Migraine Therapy Sales

 
• By 

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

Azafaros Banks Over €130m To Advance Nizubaglustat Into Phase III

 
• By 

The therapy has been developed for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.

J&J’s Imaavy Approval Is The Starting Line For Pipeline-In-A-Product Strategy

 

The company’s FcRn inhibitor nipocalimab was approved by the US FDA for generalized myasthenia gravis (gMG), where it will join a competitive market.

In Brief: Ferrer Signs Deal For Prilenia’s Pridopidine In Europe

 

The companies will co-develop the drug for additional indications beyond Huntington’s disease, while Prilenia retains rights in key global markets.

More from Scrip

Lupin CEO On Trump’s Most Favored Nation Policy, Tariffs, Firm’s Five-Year Plan

 
• By 

Lupin CEO Vinita Gupta speaks on Trump’s most favored nation policy, the Inflation Reduction Act’s “pill penalty” and other developments in the US, a major market for the company. The company also outlines a five-year plan focused on complex generics and technology platforms.

Novo Nordisk’s CEO Forced Out After Falling Behind In GLP-1 Battle

 

Company surprises investors with Jørgensen's exit but insists its strategy will not change.

In Brief: US FDA Delay Sends Biohaven Shares Down

 

The US regulator’s decision has taken Biohaven and investors by surprise, with the reason for a ‘major amendment’ status not clear.