1. Scrip
  2. >>Rare Diseases

Santhera's Transformation Should Turn Investors' Heads

DMD Drug To Be Filed With FDA Imminently

 
• By Kevin Grogan

The Swiss biotech had a tough time after the failure of idebenone for Duchenne muscular dystrophy, but CEO Dario Eklund tells Scrip that with a new soon-to-be submitted compound, vamorolone, it has an effective and much safer alternative to steroids for the treatment of the progressive muscle degeneration genetic disorder.

Eklund_Dario_1200
Dario Eklund • Source: Santhera

The scale and speed of the successful turnaround at Santhera Pharmaceuticals has gone largely unnoticed by the investment community but a restructuring and a very promising Duchenne muscular dystrophy (DMD) project suggest that the Swiss biotech's comeback is nearly complete.

The change in fortune has been led by Dario Eklund who joined Santhera as CEO in December 2019 and he...

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

Another Day Another Win For Novartis’s Ianalumab – This Time In ITP

 
• By Aakash Babu

The Swiss drugmaker’s BAFF-R inhibitor and ADCC-mediated B-cell depletor candidate has chalked up another win in late-stage development, further strengthening its pipeline-in-a-product bid.

Prasad’s Exit From CBER Could Bode Well For Genetic Medicines

 
• By Jessica Merrill

Pharma companies like Sarepta, Replimune and Capricor made gains on Wall Street after news broke that the FDA Center for Biologics Evaluation and Research director Vinay Prasad is leaving.

PTC To Challenge BioMarin In PKU With Oral Sephience

 
• By Joseph Haas

PTC plans to compete with BioMarin’s two phenylketonuria drugs with efficacy data showing strong reduction of phenylalanine and ability for patients to liberalize their diets.

Apellis’ Empaveli Expansion Underway In Rare Kidney Disease

 
• By Jessica Merrill

The US FDA approved the complement C3 inhibitor for new indications in C3G and primary IC-MPGN, providing a new growth opportunity for Apellis.

More from Scrip

Pipeline Watch: Seven Approvals And Five Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Indegene Exec On DTP Distribution As A ‘Tactical Solution’, Feasibility Of MFN Model

 
• By Anju Ghangurde

Senior Indegene executive William Lobb talks about complexities of the direct-to-patient distribution model in the US and underlines that it isn’t a comprehensive healthcare fix. The feasibility of MFN pricing and why it may be “catastrophic” to the industry were some of the other topics discussed.

Quick Listen: Scrip’s Five Must-Know Things

 
• By Ian Haydock

In this week's episode: a look at the likely top drugs in 2030; Lilly CEO advocates US/Europe pricing rebalance; Insmed sees $5bn-plus market for Brinsupri; royalty deals go mainstream; Phase III win sets stage for Epkinly label expansion.