Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50

Roche’s Evrysdi wins FDA approval in newborns with spinal muscular atrophy

By Brian Buntz | May 31, 2022

Roche in Drug Discovery & Development Pharma 50Roche (SIX:RO, ROG; OTCQX:RHHBY) has announced that the FDA has approved a label extension to allow the use of Evrysdi (risdiplam) in babies younger than two months old with spinal muscular atrophy (SMA).

SMA is a genetic disease associated with progressive muscle wasting and weakness that affects roughly 1 in 11,000 babies.

The drug first won FDA approval in 2020 for adults and children at least two months old.

The label extension was based on interim efficacy and safety data from the RAINBOWFISH study in newborns.

That data demonstrated the majority of pre-symptomatic babies who received Evrysdi achieved developmental milestones related to sitting and standing. In addition, half of the babies were walking after 12 months of treatment.

“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA before symptoms start to arise can help babies to achieve motor milestones,” said Dr. Richard Finkel, RAINBOWFISH principal investigator and director of the experimental neuroscience program at St. Jude Children’s Research Hospital. “With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed.”

Evrysdi modulates the survival of motor neuron-2 (SMN2) protein.

More than 5,000 patients have been treated with the drug to date, including babies, children and adults.

Spinal muscular atrophy results from the homozygous deletion or mutations in survival motor neuron-1 (SMN1).

Extra copies of the SMN2 gene do not cause spinal muscular atrophy, but they modify the severity of the disorder, according to NIH.


Filed Under: Neurological Disease
Tagged With: Evrysdi, FDA, risdiplam, SMN2, spinal muscular atrophy, survival of motor neuron-2
 

Tell Us What You Think! Cancel reply

You must be logged in to post a comment.

Related Articles Read More >

Astellas Pharma in the Drug Discovery & Development Pharma 50
FDA places clinical hold on Astellas clinical trial focused on Pompe disease
FDA
FDA launches 5-year initiative for rare neurodegenerative diseases 
Biogen in the Drug Discovery & Development Pharma 50
Biogen halts observational post-marketing study of aducanumab
Amylyx
Inside Canada’s approval of Amylyx’s ALS drug Albrioza

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup
Drug Discovery and Development
  • MASSDEVICE
  • DEVICETALKS
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • MEDTECH 100
  • Medical Design Sourcing
  • Subscribe to our Free E-Newsletter
  • Contact Us
  • About Us
  • Advertise With Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2022 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50