Risdiplam

Summary

Risdiplam (CHEMBL4297528) is an approved small molecule (ATC M09AX10); indicated across 3 conditions including spinal muscular atrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: M09AX10
• Indications: 3 conditions
• Clinical trials: 19
• Chemistry: 401.5 Da · C22H23N7O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297528
Name	Risdiplam
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	118513932
ATC	M09AX10
Molecular formula	C22H23N7O
Molecular weight	401.5
InChIKey	ASKZRYGFUPSJPN-UHFFFAOYSA-N

SMILES: CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6

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

Also known as: Evrysdi, RG-7916, RG7916, Risdiplam, RO-7034067, RO7034067, RISDIPLAM

Patent coverage: 158 distinct patent families (397 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
spinal muscular atrophy	4	MONDO:0001516	EFO:0008525

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 19.

Phase distribution

Phase	Trials
PHASE2	6
PHASE1	5
PHASE4	4
Not specified	3
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05232929	PHASE4	ACTIVE_NOT_RECRUITING	Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
NCT05522361	PHASE4	ACTIVE_NOT_RECRUITING	Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
NCT05861986	PHASE4	RECRUITING	A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
NCT05861999	PHASE4	RECRUITING	A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
NCT05115110	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
NCT03779334	PHASE2	ACTIVE_NOT_RECRUITING	A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
NCT05808764	PHASE2	RECRUITING	A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
NCT07047144	PHASE2	RECRUITING	A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
NCT02908685	PHASE2	COMPLETED	A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
NCT02913482	PHASE2	COMPLETED	Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
NCT03032172	PHASE2	COMPLETED	A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
NCT02633709	PHASE1	COMPLETED	A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
NCT03040635	PHASE1	COMPLETED	A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants
NCT03920865	PHASE1	COMPLETED	A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
NCT03988907	PHASE1	COMPLETED	A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
NCT04718181	PHASE1	COMPLETED	Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
NCT06978985	Not specified	RECRUITING	Adult SMA Research and Clinical Hub
NCT04256265	Not specified	APPROVED_FOR_MARKETING	An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
NCT05219487	Not specified	COMPLETED	Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).