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1825352-65-5

1825352-65-5 structural image
Product Name: Risdiplam
Formula: C22H23N7O
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SAFETY INFORMATION

Signal word Warning
Pictogram(s)

Exclamation Mark
Irritant
GHS07
GHS Hazard Statements H302:Acute toxicity,oral
H315:Skin corrosion/irritation
H319:Serious eye damage/eye irritation
H335:Specific target organ toxicity, single exposure;Respiratory tract irritation
Precautionary Statement Codes P261:Avoid breathing dust/fume/gas/mist/vapours/spray.
P305+P351+P338:IF IN EYES: Rinse cautiously with water for several minutes. Remove contact lenses, if present and easy to do. Continuerinsing.

COMPUTED DESCRIPTORS

Molecular Weight 401.5 g/mol
XLogP3 0.5
Hydrogen Bond Donor Count 1
Hydrogen Bond Acceptor Count 6
Rotatable Bond Count 2
Exact Mass 401.19640838 g/mol
Monoisotopic Mass 401.19640838 g/mol
Topological Polar Surface Area 78.1 Ų
Heavy Atom Count 30
Formal Charge 0
Complexity 886
Isotope Atom Count 0
Defined Atom Stereocenter Count 0
Undefined Atom Stereocenter Count 0
Defined Bond Stereocenter Count 0
Undefined Bond Stereocenter Count 0
Covalently-Bonded Unit Count 1
Compound Is Canonicalized Yes

PRODUCT INTRODUCTION

description

Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA). It increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription. This mechanism of action is similar to its predecessor [nusinersen], the biggest difference being their route of administration: nusinersen requires intrathecal administration, as does the one-time gene therapy [onasemnogene abeparvovec], whereas risdiplam offers the ease of oral bioavailability. Risdiplam was approved by the FDA in August 2020 for the treatment of spinal muscular atrophy (SMA). Set to be substantially cheaper than other available SMA therapies, risdiplam appears to provide a novel and relatively accessible treatment option for patients with SMA regardless of severity or type.

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