Spinal Muscular Atrophy News

Novartis presented long-term data of Zolgensma in 5+ years patients


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

The data shows that the patients 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, patients were treated pre-symptomatically and achieved motor milestones, including sitting, standing, and walking.


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

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


Also read: TGA News | TGA Approved Zolgensma


Results in two-copy cohort:

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


Results in three-copy cohort:

53% of patients could stand for 30 seconds (primary endpoint), 40% of patients 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 patients 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 in patients 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) - Patients 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 patient-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.


Also read: Pricing and market access in Netherlands


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