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5mg |
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25mg |
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Purity: ≥98%
Risdiplam (RG7916, RO7034067; Evrysdi) is a novel, potent, orally bioactive and centrally and peripherally distributed gene splicing modulator (neuromuscular disease) approved to treat spinal muscular atrophy. Risdiplam increases the levels of survival motor neuron (SMN) protein.
Targets |
SMN2
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ln Vitro |
Risdiplam raises the amounts of SMN protein and controls the splicing of SMN2 pre-mRNA to generate full-length SMN2 mRNA. Risdiplam is a splicing modulator of SMN2 that can enhance the amount of full-length SMN2 protein, enhancing the functionality of SMN proteins. The most frequent genetic illness that kills infants is still (SMA). Due to doublets in the southeastern motor neuron 1 (SMN1) gene, low levels of early motor neuron protein (SMN) are the cause of this autosomal recessive neuropathy disorder, which is characterized by progressive movement and respiratory muscle weakening. Other sources of residual inactivation and gene loss [1].
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References |
[1]. Poirier A, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447.
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Molecular Formula |
C22H23N7O
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Molecular Weight |
401.464323282242
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Exact Mass |
401.2
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Elemental Analysis |
C, 65.82; H, 5.77; N, 24.42; O, 3.99
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CAS # |
1825352-65-5
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Related CAS # |
Risdiplam-d4
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Appearance |
Solid powder
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SMILES |
CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6
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InChi Key |
ASKZRYGFUPSJPN-UHFFFAOYSA-N
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InChi Code |
InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3
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Chemical Name |
7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one
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Synonyms |
RG7916; RO703406; RG-7916; RO-7034067;RG 7916; RO 7034067; Evrysdi
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HS Tariff Code |
2934.99.9001
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Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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Solubility (In Vitro) |
DMSO: ~1.7 mg/mL (~4.2 mM)
Ethanol: < 1 mg/mL H2O: < 0.1 mg/mL |
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Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80: Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 :Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400  (Please use freshly prepared in vivo formulations for optimal results.) |
Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
1 mM | 2.4909 mL | 12.4545 mL | 24.9091 mL | |
5 mM | 0.4982 mL | 2.4909 mL | 4.9818 mL | |
10 mM | 0.2491 mL | 1.2455 mL | 2.4909 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration: mg/mL;
Method for preparing DMSO stock solution: mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation::Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O,mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
NCT Number | Recruitment | interventions | Conditions | Sponsor/Collaborators | Start Date | Phases |
NCT04888702 | Active Recruiting |
Device: Actimyo | Spinal Muscular Atrophy | Dr. Stéphanie Delstanche | August 8, 2018 | Not Applicable |
NCT05156320 | Active Recruiting |
Drug: Apitegromab Drug: Placebo |
SMA Atrophy |
Scholar Rock, Inc. | February 24, 2022 | Phase 3 |
NCT03779334 | Active Recruiting |
Drug: Risdiplam | Muscular Atrophy, Spinal | Hoffmann-La Roche | August 7, 2019 | Phase 2 |
NCT03032172 | Active Recruiting |
Drug: Risdiplam | Spinal Muscular Atrophy | Hoffmann-La Roche | March 3, 2017 | Phase 2 |
NCT02913482 | Active Recruiting |
Drug: Risdiplam | Muscular Atrophy, Spinal | Hoffmann-La Roche | December 23, 2016 | Phase 2 |
Risdiplam tissues concentration vs risdiplam plasma concentration: A, brain (n = 189) (Studies 1‐4, 6‐10). Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |
Risdiplam tissue distribution in cynomolgus monkeys. Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |
Time course of plasma and tissue risdiplam concentrations in mice or rats following PO or IP, single or repeat administration of risdiplam. Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |
SMN protein increase in muscle vs brain in SMA mice following administration of risdiplam. Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |