Details of the Drug

General Information of Drug (ID: DMIMF8J)

• Drug Name: Risdiplam
• Synonyms: UNII-76RS4S2ET1; 76RS4S2ET1; 1825352-65-5; Risdiplam [INN]; SCHEMBL17260852; EX-A2074; RG-7916; HY-109101; RO7034067; CS-0039501; 4H-Pyrido(1,2-a)pyrimidin-4-one, 7-(4,7-diazaspiro(2.5)oct-7-yl)-2-(2,8-dimethylimidazo(1,2-b)pyridazin-6-yl)-; 2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-7-(4,7-diazaspiro[2.5]octan-7-yl)-4H-pyrido[1,2-a]pyrimidin-4-one

Indication

Disease Entry	ICD 11	Status	REF
Spinal muscular atrophy	8B61	Approved	[1]

• ATC Code:
  M09AX10: Risdiplam
  M09AX: Other drugs for disorders of the musculo-skeletal system
  M09A: OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
  M09: OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
  M: MUSCULO-SKELETAL SYSTEM
• #Ro5 Violations (Lipinski): 0:
  Molecular Weight (mw); 401.5
  Logarithm of the Partition Coefficient (xlogp); 0.5
  Rotatable Bond Count (rotbonds); 2
  Hydrogen Bond Donor Count (hbonddonor); 1
  Hydrogen Bond Acceptor Count (hbondacc); 6
• ADMET Property:
  Absorption Tmax: The time to maximum plasma concentration (Tmax) is 1-4 h [2]
  Elimination: Following the oral administration of 18mg risdiplam, approximately 53% of the dose was excreted in the feces and 28% was excreted in the urine [2]
  Half-life: The concentration or amount of drug in body reduced by one-half in 50 hours [2]
  Metabolism: The drug is metabolized via the flavin monooxygenases 1 and 3 (FMO1 and FMO3), with some involvement of CYP1A1, CYP2J2, CYP3A4, and CYP3A7 [2]
  Vd: The volume of distribution (Vd) of drug is 6.3 L/kg [3]
• Chemical Identifiers:
  Formula: C22H23N7O
  IUPAC 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
  Canonical SMILES: CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6
  InChI: 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
  InChIKey: ASKZRYGFUPSJPN-UHFFFAOYSA-N
• Cross-matching ID:
  PubChem CID: 118513932
  CAS Number: 1825352-65-5
  UNII: 76RS4S2ET1
  DrugBank ID: DB15305
  TTD ID: D0N5MH
  INTEDE ID: DR1823
  ACDINA ID: D01397

Molecular Interaction Atlas of This Drug

Drug-Metabolizing Enzyme (DME)

DME Name	DME ID	UniProt ID	MOA	REF
Cytochrome P450 3A4 (CYP3A4) Main DME	DE4LYSA	CP3A4_HUMAN	Substrate	[4]

Drug Inactive Ingredient(s) (DIG) and Formulation(s) of This Drug

DIG

DIG Name	DIG ID	PubChem CID	Functional Classification
Isomalt	E00383	88735	Binding agent; Coating agent; Diluent; Flavoring agent; Suspending agent
Mannitol	E00103	6251	Diluent; Flavoring agent; Lyophilization aid; Plasticizing agent; Tonicity agent
Sodium benzoate	E00432	517055	Antimicrobial preservative; lubricant
Sucralose	E00370	71485	Flavoring agent
Ascorbic acid	E00579	54670067	Acidulant; Antioxidant
Edetate disodium	E00186	8759	Complexing agent
Polyethylene glycol 6000	E00655	Not Available	Coating agent; Diluent; Ointment base; Plasticizing agent; Solvent; Suppository base; lubricant
Tartaric acid	E00029	875	Acidulant; Complexing agent; Flavoring agent

Pharmaceutical Formulation

Formulation Name	Drug Dosage	Dosage Form	Route
Risdiplam 0.75 mg/ml powder	0.75 mg/ml	Powder	Oral

Jump to Detail Pharmaceutical Formulation Page of This Drug

References

• [1] Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health Human Services. 2020
• [2] FDA Approved Drug Products: Evrysdi (risdiplam) powder for oral solution
• [3] Ramdas S, Servais L: New treatments in spinal muscular atrophy: an overview of currently available data. Expert Opin Pharmacother. 2020 Feb;21(3):307-315. doi: 10.1080/14656566.2019.1704732.
• [4] A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier. Br J Clin Pharmacol. 2019 Jan;85(1):181-193.