Definitive Data Hopes Keep Santhera's Duchenne Drug Alive in UK Despite EMA No

The European Medicines Agency rejected Santhera’s Raxone as a treatment for Duchenne muscular dystrophy but the prospect of definitive data supporting the new indication means idebenone will still be available to patients in the UK under the early access scheme for promising new medicines. 

Duchenne
Santhera's DMD treatment remains on the UK early access scheme • Source: Shutterstock

The UK drug regulator has decided not to drop Santhera Pharmaceuticals’s drug Raxone (idebenone) from its early access to medicines scheme (EAMS) as a treatment for Duchenne muscular dystrophy, despite the European Medicines Agency saying the product should not be approved.

The EAMS designation for Raxone was granted last June and allows Santhera to make the drug available to patients in...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet 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 Market Access

US Pricing Reform: New Products Offer ‘On Ramp’ To Implementation, Lilly’s Ricks Says

 
• By 

Eli Lilly's CEO argued that pricing reforms aimed at existing drugs would be “hard to sustain or justify,” while other companies suggested a growing interest in direct-to-consumer sales programs.

EU Must Up Competitiveness To Counter US Tariffs And Pricing Woes For SMEs

 

Companies in Europe cannot delay business decision making much longer to wait for more certainty on major US policy shifts on tariffs and pricing.

50% Levy On India And Brazil, US Tariffs Leave Global Trade, Forecasts In Disarray

 
• By 

50% tariffs on US imports from India, a range on others, an uncertain outcome of Section 232 investigations of pharma and talk of BRICS tariffs are making the forecasting environment for the pharma industry extremely difficult, the Pink Sheet finds in this infographic analysis.

England: Pharma Firms To Benefit From Parallel HTA And Regulatory Pathway

 

The UK drug regulator and England’s health technology assessment agency have joined forces under an information sharing agreement, aimed at accelerating patient access to newly approved medicines by three to six months.

More from Pink Sheet

Global Pharma Guidance Tracker - July 2025

 

Stay up to date on regulatory guidelines from around the world with the Pink Sheet’s Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.

Research Advances And Filings Prompt EMA To Revise Alzheimer’s Trial Guideline

 

Following EU approval of the first disease-modifying treatment for Alzheimer’s disease, and with a second expected soon, the European Medicines Agency plans to update its guidance to support the design of state-of-the-art clinical trials for the disease and enhance drug development.

50% Levy On India And Brazil, US Tariffs Leave Global Trade, Forecasts In Disarray

 
• By 

50% tariffs on US imports from India, a range on others, an uncertain outcome of Section 232 investigations of pharma and talk of BRICS tariffs are making the forecasting environment for the pharma industry extremely difficult, the Pink Sheet finds in this infographic analysis.