Chugai Receives Japanese Indication Approval For Evrysdi In Pre-Symptomatic SMA

Chugai Receives Japanese Indication Approval For Evrysdi In Pre-Symptomatic SMA

By, Admin 26 September 2024

Chugai receives Japanese additional indication approval for Evrysdi in pre-symptomatic SMA and additional dosage for infants under 2 months of age

Overview

Chugai Pharmaceutical Co., Ltd. announced that the Ministry of Health, Labour and Welfare (MHLW) has approved today “Evrysdi dry syrup 60 mg” (risdiplam) for an additional indication of pre-symptomatic spinal muscular atrophy (SMA) predicted by genetic testing, and an additional dosage for patients under 2 months of age. Evrysdi received orphan drug designation from MHLW for “spinal muscular atrophy” in March 2019, and the application for additional indication and dosage were approved under priority review.

Statement from the President: Chugai

  • We are very pleased that with this approval, we can now deliver Evrysdi to people with SMA of all ages after birth, regardless of symptom onset. As the only oral treatment for SMA, Evrysdi has been helping many people with SMA and their families.
  • SMA treatment is expected to maximize its effect when intervention begins at an earlier stage. We are confident that this approval will allow Evrysdi to contribute even more to SMA treatment” said Chugai’s president and CEO, Dr. Osamu Okuda.

Behind the Approval

  • This approval is based on the results of the overseas phase II RAINBOWFISH study, for pre-symptomatic SMA infants (up to 6 weeks of age at first dose) diagnosed genetically with SMA. 
  • The RAINBOWFISH study included infants with two or more copies of the SMN2 gene. 
  • Generally, the lower the number, the more severe the disease.

Early Screening of SMA

  •  In SMA, the loss of motor neurons may begin before symptoms start, so initiating treatment early is critical for better outcomes, and newborn screening plays an important role for early diagnosis. 
  • Evrysdi is expected to provide high medical value by enabling immediate treatment after diagnosis, regardless of symptom onset.

The RAINBOWFISH Trial

  • The RAINBOWFISH study [NCT03779334] is an open-label, single-arm, multicentre study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. 
  • The primary endpoint was the proportion of infants in the primary efficacy population who are sitting without support for at least 5 seconds at month 12 assessed by the BSID-III (Bayley Scales of Infant and Toddler Development - Third Edition) gross motor scale. 
  • Japan is not included in this study.

About Evrysdi

  • Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. 
  • Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein. 
  • SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. 
  • Evrysdi was approved in the US in August 2020, in Europe in March 2021, and in Japan in June 2021. 
  • In Japan, it has now received approval for an additional indication in pre-symptomatic SMA and an additional dosage for infants under 2 months of age.

Requirement for Additional Tablet Formulation

  • Additionally, an application for the addition of a tablet formulation (for patients 2 years of age and older weighing 20 kg or more) was submitted in April 2024 as a new treatment option. 
  • If approved, the tablet formulation will allow for storage and transport at room temperature and eliminate the need for measuring doses.
  • It leads to more convenient storage and administration, enabling contribution for treatment choices that suit individual conditions and lifestyles.

The Disorder: Spinal Muscular Atrophy

  • Spinal muscular atrophy (SMA) is a genetic neuromuscular disease that causes muscule atrophy and muscle weakness due to degeneration of the motor neuron. 
  • The causative gene for SMA is the survival motor neuron (SMN) gene.
  • The disease develops because of insufficient production of functional SMN protein from SMN2 gene alone, in addition to the dysfunction of the SMN1 gene.

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