- Roche Holdings AG RHHBY announced new two-year data from the JEWELFISH study evaluating Evrysdi (risdiplam) in people with Type 1, 2, or 3 spinal muscular atrophy (SMA) aged six months to 60 years.
- Patients had been previously treated with other approved or investigational SMA-targeting therapies, including Biogen Inc's BIIB Spinraza (nusinersen) or Novartis AG's NVS Zolgensma (onasemnogene abeparvovec).
- Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels sustained after two years of treatment.
- Related: Roche Releases More Evrysdi Data For Very Young Infants With SMA.
- People with SMA cannot produce enough survival motor neuron (SMN) protein.
- The study showed Evrysdi led to a two-fold increase in median SMN protein levels versus baseline after four weeks of treatment in all patient groups, irrespective of previous treatment.
- The SMN protein levels achieved after four weeks of treatment were maintained for over two years.
- Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics Inc PTCT.
- Price Action: RHHBY shares closed higher by 1.40% at $40.53 on Tuesday.
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