Risdiplam

Risdiplam
Clinical data
Other names	RG7916; RO7034067
ATC code	None;
Identifiers
	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;
CAS Number	1825352-65-5;
PubChem CID	118513932;
UNII	76RS4S2ET1;
KEGG	D11406;
Chemical and physical data
Formula	C22H23N7O
Molar mass	401.474 g·mol−1
3D model (JSmol)	Interactive image;
	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; Key:ASKZRYGFUPSJPN-UHFFFAOYSA-N;

Risdiplam (RG7916, RO7034067) is an experimental drug being developed by F. Hoffmann-La Roche, PTC Therapeutics and SMA Foundation to treat spinal muscular atrophy (SMA). It appears to be effective orally and is a small molecule. It is a pyridazine derivative that works by increasing the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.[1][2]

Risdiplam has shown good safety and efficacy in phase II–III clinical trials.[3][4] As of April 2020, Roche has applied for marketing authorisation in Brazil, Chile, Indonesia, Russia, South Korea, Taiwan and the United States while applications in China and the European Union are to be submitted by mid-2020.[5]

Since late 2019, Roche has been offering the drug globally for free for eligible people through an expanded access programme.[6]

References

Maria Joao Almeida (2016-09-08). "RG7916". BioNews Services. Retrieved 2017-10-08.
Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, et al. (May 2016). "Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy". Human Molecular Genetics. 25 (10): 1885–1899. doi:10.1093/hmg/ddw062. PMC 5062580. PMID 26931466.
Baranello, G.; Servais, L.; Day, J.; Deconinck, N.; Mercuri, E.; Klein, A.; Darras, B.; Masson, R.; Kletzl, H.; Cleary, Y.; El-Khairi, M. (2019-10-01). "P.353FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with type 1 spinal muscular atrophy". Neuromuscular Disorders. 29: S184. doi:10.1016/j.nmd.2019.06.515. ISSN 0960-8966.
Mercuri, Eugenio; Baranello, Giovanni; Kirschner, Janbernd; Servais, Laurent; Goemans, Nathalie; Pera, Maria Carmela; Buchbjerg, Jeppe; Yeung, Wai Yin; Kletzl, Heidemarie; Gerber, Marianne; Czech, Christian (2019-04-09). "Update from SUNFISH Part 1: Safety, Tolerability and PK/PD from the Dose-Finding Study, Including Exploratory Efficacy Data in Patients with Type 2 or 3 Spinal Muscular Atrophy (SMA) Treated with Risdiplam (RG7916) (S25.007)". Neurology. 92 (15 Supplement). ISSN 0028-3878.
"Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)". www.roche.com. Retrieved 2020-04-08.
"Roche announces global compassionate use programme for risdiplam". Spinal Muscular Atrophy UK. Retrieved 2020-04-08.

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[1] Maria Joao Almeida (2016-09-08). "RG7916". BioNews Services. Retrieved 2017-10-08.

[2] Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, et al. (May 2016). "Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy". Human Molecular Genetics. 25 (10): 1885–1899. doi:10.1093/hmg/ddw062. PMC 5062580. PMID 26931466.

[3] Baranello, G.; Servais, L.; Day, J.; Deconinck, N.; Mercuri, E.; Klein, A.; Darras, B.; Masson, R.; Kletzl, H.; Cleary, Y.; El-Khairi, M. (2019-10-01). "P.353FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with type 1 spinal muscular atrophy". Neuromuscular Disorders. 29: S184. doi:10.1016/j.nmd.2019.06.515. ISSN 0960-8966.

[4] Mercuri, Eugenio; Baranello, Giovanni; Kirschner, Janbernd; Servais, Laurent; Goemans, Nathalie; Pera, Maria Carmela; Buchbjerg, Jeppe; Yeung, Wai Yin; Kletzl, Heidemarie; Gerber, Marianne; Czech, Christian (2019-04-09). "Update from SUNFISH Part 1: Safety, Tolerability and PK/PD from the Dose-Finding Study, Including Exploratory Efficacy Data in Patients with Type 2 or 3 Spinal Muscular Atrophy (SMA) Treated with Risdiplam (RG7916) (S25.007)". Neurology. 92 (15 Supplement). ISSN 0028-3878.

[5] "Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)". www.roche.com. Retrieved 2020-04-08.

[6] "Roche announces global compassionate use programme for risdiplam". Spinal Muscular Atrophy UK. Retrieved 2020-04-08.