Roche Sticking With SMA Despite Terminating Olesoxime

Problems over formulation and the arrival of effective treatment options in spinal muscular atrophy, headed by Biogen's antisense oligonucleotide drug Spinraza, have led to the plug being pulled on olesoxime - but the Swiss major has a back-up candidate.

SC1806_Broken Light Bulb_686792284_1200.jpg
One project blown, one left for Roche in SMA • Source: Shutterstock

The emergence of effective treatments for spinal muscular atrophy and "many difficulties" in its development has led Roche to axe olesoxime, which was due to start Phase III trials later this year for the rare genetic disorder.

The Swiss major got hold of olesoxime three years ago when it acquired France's Trophos, paying €120m up front, 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

More from Rare Diseases

More from Scrip

Compass Pathways’ Antidepressant Advances In An Increasingly Tough Psychedelic Market

 
• By 

Psychedelics appear to be in vogue after years of being relegated to alternative medicine. But investor pushback after the highly anticipated release of Phase III trial data by Compass Pathways reveals an increasingly high bar for development.

Eolo Pharma Advances In Obesity With Thermogenesis Approach

 
• By 

Emerging Company Profile: Early-stage biotech Eolo will seek cash and deals for its novel obesity drug SANA after positive Phase I results for weight loss, lean muscle mass preservation and fat mass reduction.

BioMarin’s Roctavian Continues to Shine In Hemophilia A Despite Commercial Woes

 

Roctavian shows durable bleed control and safety over five years, but high costs and access hurdles limit its commercial outlook.