Spinal Muscular Atrophy News

April 29, 2022

Evrysdi improved survival and motor milestones in infants with type 1 SMA

Roche announced that Evrysdi (risdiplam) improved survival and motor milestones in infants with type 1 spinal muscular atrophy (SMA).

In the FIREFISH study, 91% of infants who were treated with Evrysdi were alive after three years. The infants showed improvement in motor functions, ability to swallow, sit without support, and stand with support between two to three years.


In the FIREFISH, there are two parts, Part-1 includes identifying the dose, and Part-2 evaluates the safety and efficacy of the dose determined in Part-1.

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Novartis presented new data of Zolgensma; demonstrating efficacy in presymptomatic and symptomatic infants with SMA

Novartis presented the data of Zolgensma (onasemnogene abeparvovec) in presymptomatic and symptomatic infants with spinal muscular atrophy (SMA). SPR1NT is Phase 3, an open-label, single-arm study.


Also read: Denmark Healthcare System


All children who are presymptomatic in SPR1NT two-copy cohort survived with respiratory or nutritional support. Eleven out of fourteen infants with SMA could sit independently for ≥30 seconds, which falls within the WHO window of normal development.


Also read: NICE assessment outcomes


Among the symptomatic children, 82% of children developed a motor function that was not observed in the natural history of SMA Type 1. 49% of children (16 children) could sit for ≥30 seconds.


The safety profile was similar to the previously reported profile.


Novartis presented long-term data of Zolgensma in 5+ years infants with SMA


Novartis presented the new data of Zolgensma (onasemnogene abeparvovec), demonstrating real-world benefit after five years of treatment.

The data shows that infants with SMA treated pre-symptomatically achieved age-related milestones, including sitting, walking, requiring no ventilatory or feeding tube support. The results were presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference.


Also read: Denmark Healthcare System



In Phase 3 SPR1NT trial, infants with SMA were treated pre-symptomatically and achieved motor milestones, including sitting, standing, and walking.


Infants with two copies of SMN2 develop SMA Type 1, and infants with three copies of SMN2 suffer from SMA Type 2.

Across the two-copy and three-copy cohorts, 100% of infants were free of ventilatory and feeding support.


Also read: TGA News | TGA Approved Zolgensma


Results in two-copy cohort:

79% of infants with SMA were able to sit without support for 30 seconds (primary endpoint). Five infants were able to stand, three of them met the WHO window of normal development. Four infants were able to walk, three of them were able to meet the WHO window of normal development. 100% of infants achieved CHOP INTEND scores of ≥50, and 93% of infants achieved CHOP INTEND scores of ≥58.


Results in three-copy cohort:

53% of infants could stand for 30 seconds (primary endpoint), 40% of infants walked independently.


Also read: The United States Healthcare System


Novartis has reported no significant adverse events in the long term.

 

Roche presented 2-year data of Evrysdi, which shows improvement in motor function in infants with SMA Type 2 or Type 3

Roche has presented the 2-year long-term study of Evrysdi (risdiplam). The company has presented the results of infants with SMA of age 2-25 years. The study suggests that the motor gains achieved at the 12th month were continued for 24 months across primary and secondary endpoints. The data was presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference.

Also read: Denmark Healthcare System



In the SUNFISH Part 2 study, the drug is compared with placebo.

  • Motor functions measured using Motor Function Measure (MGM-32) - Infants with SMA treated with Evrysdi improved motor function at month 12 and 24

  • Motor functions measured by Revised Upper Limb Module (RULM) and the Hammersmith Functional Motor Scale-Expanded (HFMSE) - Motor function was increased between months 12 and 24

  • Motor functions were stabilized after the treatment with Evrysdi, measured by MFM-32, RULM, and HFMSE

  • Caregiver-reported SMAIS upper limb module and the infants-reported SMAIS score increased from baseline between 12th month and 24th month

The common adverse events reported were pneumonia, influenza, respiratory tract infections, nasopharyngitis, pyrexia, headache, diarrhea, vomiting, and cough.


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