Roche's Muscular Atrophy Treatment Improves Motor Function In Pretreated Patients

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.