FDA approves Roche?s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older
- In two clinical trials, Evrysdi improved motor function in people living with SMA over a broad spectrum of ages and levels of disease severity, including Types 1, 2, and 3 SMA
- Evrysdi helped infants survive without permanent ventilation and achieve the ability to sit without support, a key motor milestone not normally seen in the natural course of the disease
- Evrysdi is the first and only medicine for SMA that can be taken at home
- 41% (7/17) of infants treated with the therapeutic dose achieved the ability to sit without support for at least 5 seconds as measured by the BSID-III gross motor scale.
- 90% (19/21) of all infants were alive without permanent ventilation* and reached 15 months of age or older
- 81% (17/21) of all patients were alive without permanent ventilation* after a minimum of 23 months of treatment and reached an age of 28 months or older (median 32 months; range 28 to 45 months)
- A clinically-meaningful and statistically significant improvement in motor function among children and adults, as measured by a change from baseline in the MFM-32 total score (1.55 point mean difference; p=0.0156), at 12 months as compared to placebo (1.36 points [95% CI: 0.61, 2.11]; -0.19 points [95% CI: 1.22, 0.84], respectively). MFM-32 assesses 32 different motor functions across a wide range of people with SMA.
- Improved upper limb motor function compared to baseline, as measured by the Revised Upper Limb Module (RULM), a secondary independent motor function endpoint of the study (1.59 point difference; p=0.0028).
- JEWELFISH (NCT03032172): an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. Recruitment for this study is complete with 174 people enrolled.
- RAINBOWFISH (NCT03779334): an open-label, single-arm, multicenter study investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in infants (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is currently recruiting.
Dr. Nicolas Dunant Phone: +41 61 687 05 17 | Patrick Barth Phone: +41 61 688 44 86 |
Daniel Grotzky Phone: +41 61 688 31 10 | Karsten Kleine Phone: +41 61 682 28 31 |
Nina M?hlitz Phone: +41 79 327 54 74 | Nathalie Meetz Phone: +41 61 687 43 05 |
Barbara von Schnurbein Phone: +41 61 687 89 67 |
Roche Investor Relations | |
Dr. Karl Mahler Phone: +41 61 68-78503 e-mail:?karl.mahler@roche.com | Jon Kaspar Bayard Phone: +41 61 68-83894 e-mail:?jon_kaspar.bayard@roche.com |
Dr. Sabine Borngr?ber Phone: +41 61 68-88027 e-mail:?sabine.borngraeber@roche.com | Dr. Bruno Eschli Phone: +41 61 68-75284 e-mail:?bruno.eschli@roche.com |
Dr. Birgit Masjost Phone: +41 61 68-84814 e-mail:?birgit.masjost@roche.com | Dr. Gerard Tobin Phone: +41 61 68-72942 e-mail:?gerard.tobin@roche.com |
Investor Relations North America | |
Loren Kalm Phone: +1 650 225 3217 e-mail:?kalm.loren@gene.com | Dr. Lisa Tuomi Phone: +1 650 467 8737 e-mail:?tuomi.lisa@gene.com |
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