US FDA Puts IT Zolgensma Studies On Partial Clinical Hold

Zolgensma And IV Studies Unaffected

The roller coaster ride for Novartis with its high-profile gene therapy Zolgensma continues as the FDA places a partial hold on trials of an intrathecal version aimed at older SMA patients on the back of nerve inflammation seen in a preclinical study.

Roller coaster
Zolgensma taking Novartis on a wild ride • Source: Shutterstock

A year of highs and lows for Novartis AG’s gene therapy product Zolgensma continues with the US Food and Drug Administration now placing on partial clinical hold studies looking at intrathecal administration of the product following troubling results from an animal study.

More from Business

Boehringer Still Sees Plenty Of Juice In Jardiance

 
• By 

Strong sales growth for the German group’s SGLT2 inhibitor in 2024

Sumitomo Offloads Asia Pharma Ops To Marubeni For $480m

 
• By 

The latest in a long line of restructuring measures will see Sumitomo Pharma making a stepped sale of its pharma operations in Asia to major Japanese trading house Marubeni.

AIRNA To Take RNA Editing Into The Clinic With $155m Series B Round

 
• By 

CEO Kris Elverum told Scrip about the start-up’s platform for editing RNA to correct genetic variants that cause harm and to reproduce healthy variants as a means of treating disease.

Will They Or Won’t They? Tariff Question Looms For Drugmakers

 

Pharma executives and investors are waiting with bated breath to find out if President Trump will include drugs in a new round of tariffs to be announced on 2 April.

More from Scrip

Axsome Plans Excessive Sleepiness Study In Depression After Phase III Failure

 

The study’s failure in the overall MDD population was not a surprise, but solriamfetol’s efficacy in MDD with EDS provided a rationale for testing it in that subpopulation.

Will They Or Won’t They? Tariff Question Looms For Drugmakers

 

Pharma executives and investors are waiting with bated breath to find out if President Trump will include drugs in a new round of tariffs to be announced on 2 April.

Cerevance To Focus On Parkinson’s Adjunctive Therapy After Phase II Miss

 
• By 

After failing a Phase II monotherapy study in early Parkinson’s, Cerevance will focus on adjunctive therapy without abandoning the monotherapy concept.