View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. News
February 25, 2021updated 12 Jul 2022 11:22am

Roche reports positive data from SMA treatment trial

Roche has reported positive data from the dose finding Part I FIREFISH study of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA).

Roche has reported positive data from the dose finding Part I FIREFISH study of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA).

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed for treating SMA by boosting SMN protein production. The protein is seen all over the body and is vital to maintaining healthy motor neurons and movement.

A severe, progressive neuromuscular disease, SMA is a major genetic cause of infant mortality.

FIREFISH, an open-label, two-part pivotal study was designed to assess the safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of Evrysdi in patients aged one to seven months with Type 1 SMA.

Part 1 was a dose-escalation study that enrolled 21 infants with the primary goal of evaluating the safety profile of Evrysdi in the subjects and dose determination for Part 2.

The Part 2 single-arm study enrolled 41 infants who received treatment for two years, followed by an open-label extension.

Evaluating efficacy as measured by the proportion of infants sitting without support after 12 months of treatment was the primary objective of Part 2.

According to the latest data, Evrysdi treatment at 12 months helped 90% of infants survive without needing permanent ventilation and 33% sit without support for at least five seconds.

Furthermore, it raised the survival levels of motor neuron (SMN) protein by a median 1.9-fold from baseline in the high-dose group at 12 months.

Roche Global Product Development chief medical officer and head Levi Garraway said: “Since Evrysdi was FDA approved in August, we have been inspired by the stories and sense of hope that we have heard from people living with SMA and their families about the impact Evrysdi has had in their lives.

“The publication of the data in the New England Journal of Medicine reinforces the value of Evrysdi as an important treatment option for SMA.”

Last September, Roche reported that Evrysdi lowered the mortality rate in infants aged two to seven months with symptomatic type 1 SMA in the FIREFISH trial.

Related Companies

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Clinical Trials Arena