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1. 7-(4,7-diazaspiro(2.5)oct-7-yl)-2-(2,8-dimethylimidazo(1,2-b)pyridazin-6-yl)-4h-pyrido(1,2-a)pyrimidin-4-one
1. 1825352-65-5
2. Rg7916
3. Evrysdi
4. Ro7034067
5. Risdiplam [inn]
6. Risdiplam [usan]
7. Rg-7916
8. 76rs4s2et1
9. Ro-7034067
10. 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
11. 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
12. 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)-
13. Evrysdi (tn)
14. Risdiplam; Rg7916
15. Risdiplam [jan]
16. Risdiplam [mi]
17. Risdiplam [usan:inn]
18. Risdiplam [who-dd]
19. Unii-76rs4s2et1
20. Risdiplam (jan/usan/inn)
21. Risdiplam [orange Book]
22. Chembl4297528
23. Schembl17260852
24. Gtpl11170
25. Dtxsid701109185
26. Amy23728
27. Ex-a2074
28. Mfcd31657372
29. Who 10614
30. Compound 1 [pmid: 30044619]
31. Db15305
32. Ac-36304
33. Br166842
34. Rg7916;ro7034067
35. Hy-109101
36. Cs-0039501
37. D11406
38. F53623
39. Q48969152
40. (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
41. 7-(4,7-diazaspiro(2.5)oct-7-yl)-2-(2,8-dimethylimidazo(1,2-b)pyridazin-6-yl)-4h-pyrido(1,2-a)pyrimidin-4-one
Molecular Weight | 401.5 g/mol |
---|---|
Molecular Formula | C22H23N7O |
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 |
Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.
Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
Risdiplam helps to alleviate symptoms of spinal muscular atrophy by stimulating the production of a critical protein in which these patients are deficient. Early trials with risdiplam demonstrated up to a 2-fold increase in SMN protein concentration in SMA patients after 12 weeks of therapy.
Neuromuscular Agents
Drugs used for their actions on skeletal muscle. Included are agents that act directly on skeletal muscle, those that alter neuromuscular transmission (NEUROMUSCULAR BLOCKING AGENTS), and drugs that act centrally as skeletal muscle relaxants (MUSCLE RELAXANTS, CENTRAL). Drugs used in the treatment of movement disorders are ANTI-DYSKINESIA AGENTS. (See all compounds classified as Neuromuscular Agents.)
M09AX10
M - Musculo-skeletal system
M09 - Other drugs for disorders of the musculo-skeletal system
M09A - Other drugs for disorders of the musculo-skeletal system
M09AX - Other drugs for disorders of the musculo-skeletal system
M09AX10 - Risdiplam
Absorption
The Tmax following oral administration is approximately 1-4 hours. Following once-daily administration with a morning meal (or after breastfeeding), risdiplam reaches steady-state in approximately 7-14 days. The pharmacokinetics of risdiplam were found to be approximately linear between all studied dosages in patients with SMA.
Route of 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. Unchanged parent drug comprised 14% of the dose excreted in feces and 8% of the dose excreted in urine.
Volume of Distribution
Following oral administration, risdiplam distributes well into the central nervous system and peripheral tissues. The apparent volume of distribution at steady-state is 6.3 L/kg.
Clearance
For a 14.9kg patient, the apparent clearance of risdiplam is 6.3 L/kg.
The metabolism of risdiplam is mediated primarily by flavin monooxygenases 1 and 3 (FMO1 and FMO3), with some involvement of CYP1A1, CYP2J2, CYP3A4, and CYP3A7. Parent drug comprises approximately 83% of circulating drug material. A pharmacologically-inactive metabolite, M1, has been identified as the major circulating metabolite - this M1 metabolite has been observed _in vitro_ to inhibit MATE1 and MATE2-K transporters, similar to the parent drug.
The terminal elimination half-life of risdiplam is approximately 50 hours in healthy adults.
Spinal muscular atrophy (SMA) is a severe and progressive congenital neuromuscular disease resulting from mutations in the survival of motor neuron 1 (_SMN1_) gene responsible for making SMN proteins. Clinical features of SMA include degeneration of motor neurons in the spinal cord which ultimately leads to muscular atrophy and, in some cases, loss of physical strength. SMN proteins are expressed ubiquitously throughout the body and are thought to hold diverse intracellular roles in DNA repair, cell signaling, endocytosis, and autophagy. A secondary _SMN_ gene (_SMN2_) can also produce SMN proteins, but a small nucleotide substitution in its sequence results in the exclusion of exon 7 during splicing in approximately 85% of the transcripts - this means that only ~15% of the SMN proteins produced by _SMN2_ are functional, which is insufficient to compensate for the deficits caused by _SMN1_ mutations. Emerging evidence suggests that many cells and tissues are selectively vulnerable to reduced SMN concentrations, making this protein a desirable target in the treatment of SMA. Risdiplam is an mRNA splicing modifier for _SMN2_ that increases the inclusion of exon 7 during splicing, which ultimately increases the amount of functional SMN protein produced by _SMN2_. It does so by binding to two sites in _SMN2_ pre-mRNA: the 5' splice site (5'ss) of intron 7 and the exonic splicing enhancer 2 (ESE2) of exon 7.
Biophore is a research-driven global pharmaceutical company focused on niche APIs for the generic industry.
GDUFA
DMF Review : Reviewed
Rev. Date : 2024-05-07
Pay. Date : 2024-04-25
DMF Number : 39602
Submission : 2024-03-08
Status : Active
Type : II
Metrochem has been delivering customized volume & quality products to customers across the world, taking utmost care of their needs.
GDUFA
DMF Review : N/A
Rev. Date :
Pay. Date :
DMF Number : 40472
Submission : 2024-09-23
Status : Active
Type : II
GDUFA
DMF Review : N/A
Rev. Date :
Pay. Date :
DMF Number : 36897
Submission : 2022-04-07
Status : Active
Type : II
GDUFA
DMF Review : N/A
Rev. Date :
Pay. Date :
DMF Number : 39255
Submission : 2024-06-19
Status : Active
Type : II
GDUFA
DMF Review : Reviewed
Rev. Date : 2024-05-28
Pay. Date : 2024-03-22
DMF Number : 39755
Submission : 2024-04-30
Status : Active
Type : II
Biophore is a research-driven global pharmaceutical company focused on niche APIs for the generic industry.
GDUFA
DMF Review : Complete
Rev. Date : 2024-05-07
Pay. Date : 2024-04-25
DMF Number : 39602
Submission : 2024-03-08
Status : Active
Type : II
GDUFA
DMF Review : N/A
Rev. Date :
Pay. Date :
DMF Number : 36897
Submission : 2022-04-07
Status : Active
Type : II
GDUFA
DMF Review : Complete
Rev. Date : 2024-05-28
Pay. Date : 2024-03-22
DMF Number : 39755
Submission : 2024-04-30
Status : Active
Type : II
GDUFA
DMF Review : N/A
Rev. Date :
Pay. Date :
DMF Number : 40472
Submission : 2024-09-23
Status : Active
Type : II
GDUFA
DMF Review : N/A
Rev. Date :
Pay. Date :
DMF Number : 39255
Submission : 2024-06-19
Status : Active
Type : II
Details:
Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: Phase IIProduct Type: Small molecule
Sponsor: Chugai Pharmaceutical
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 14, 2024
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Partner/Sponsor/Collaborator : Chugai Pharmaceutical
Deal Size : Not Applicable
Deal Type : Not Applicable
Evrysdi Two-Year Data in SMA: Most Children Can Sit, Stand, and Walk Independently
Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
October 14, 2024
Details:
Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: Phase IIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 15, 2024
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Chugai Files for Evrysdi Additional Indication for Pre-Symptomatic SMA in Infants-2 Months
Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
February 15, 2024
Details:
Under the agreement, Royalty Pharma acquires additional royalties on Evrysdi (risdiplam), a survival motor neuron 2 splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Royalty Pharma
Deal Size: $1,500.0 million Upfront Cash: $1,000.0 million
Deal Type: Agreement October 19, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Royalty Pharma
Deal Size : $1,500.0 million
Deal Type : Agreement
PTC Therapeutics Announces Evrysdi® Royalty Agreement with Royalty Pharma for Up To $1.5 Billion
Details : Under the agreement, Royalty Pharma acquires additional royalties on Evrysdi (risdiplam), a survival motor neuron 2 splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : $1,000.0 million
October 19, 2023
Details:
Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency, which is used for babies under two months old with spinal muscular atrophy.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 04, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Majority of Newborn Babies With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi A...
Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency, which is used for babies under two months old with spinal mu...
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
October 04, 2023
Details:
Evrysdi (risdiplam) is the only non-invasive spinal muscular atrophy therapy approved to treat people of all ages in the European Union now including babies from birth, Approval was based on interim data from ongoing RAINBOWFISH trial.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 29, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Evrysdi (risdiplam) is the only non-invasive spinal muscular atrophy therapy approved to treat people of all ages in the European Union now including babies from birth, Approval was based on interim data from ongoing RAINBOWFISH trial.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
August 29, 2023
Details:
Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency, which is used for babies under two months old with spinal muscular atrophy.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 29, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency, which is used for babies under two months old with spinal mu...
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
August 29, 2023
Details:
Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: Phase IVProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 21, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
CHMP Recommends Roche’s Evrysdi for Babies Under Two Months Old with Spinal Muscular Atrophy (SM...
Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
July 21, 2023
Details:
Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: Phase IVProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 30, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Four-Year Follow up Data for Roche’s Evrysdi Show Continued Increase in Number of Children with ...
Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
June 30, 2023
Details:
Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 20, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
March 20, 2023
Details:
The study showed Evrysdi (risdiplam) led to a two-fold increase in median SMN protein levels versus baseline after 4 weeks of treatment in all patient groups, irrespective of previous treatment.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Brand Name: Evrysdi
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 12, 2022
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Positive New Data For Roche’s Evrysdi In Largest Trial Ever Undertaken In Patients With Previous...
Details : The study showed Evrysdi (risdiplam) led to a two-fold increase in median SMN protein levels versus baseline after 4 weeks of treatment in all patient groups, irrespective of previous treatment.
Brand Name : Evrysdi
Molecule Type : Small molecule
Upfront Cash : Not Applicable
October 12, 2022
7-fluoro-2-hydroxy-4H-pyrido[1,2-a]pyrimidin-4-one
CAS Number : 1449598-85-9
End Use API : Risdiplam
About The Company : LinkChem is a leading China headquartered CMO | CRO provider within the pharmaceutical industry. Our core focus includes: custom synthesis, process development,...
4-Boc-4,7-diazaspiro[2.5]octane
CAS Number : 674792-08-6
End Use API : Risdiplam
About The Company : LinkChem is a leading China headquartered CMO | CRO provider within the pharmaceutical industry. Our core focus includes: custom synthesis, process development,...
2,8-Dimethyl-6-(4,4,5,5-tetramethyl-1,3,2-dioxabor...
CAS Number : 1825352-86-0
End Use API : Risdiplam
About The Company : LinkChem is a leading China headquartered CMO | CRO provider within the pharmaceutical industry. Our core focus includes: custom synthesis, process development,...
Regulatory Info : Prescription
Registration Country : Canada
Brand Name : EVRYSDI
Dosage Form : POWDER FOR SOLUTION
Dosage Strength : 0.75MG/ML
Packaging :
Approval Date :
Application Number : 2514931
Regulatory Info : Prescription
Registration Country : Canada
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Brand Name : Evrysdi
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Registration Country : Australia
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Registration Country : Australia
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Brand Name : Evrysdi
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Registration Country : Australia
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Registration Country : Australia
Brand Name : Evrysdi
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Registration Country : Australia
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Registration Country : Australia
Brand Name : Evrysdi
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Registration Country : Australia
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Registration Country : Australia
Brand Name : Evrysdi
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Registration Country : Australia
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Registration Country : Australia
Brand Name : Evrysdi
Dosage Form :
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Packaging : 1
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Application Number :
Regulatory Info :
Registration Country : Australia
RLD : Yes
TE Code :
Brand Name : EVRYSDI
Dosage Form : FOR SOLUTION;ORAL
Dosage Strength : 0.75MG/ML
Approval Date : 2020-08-07
Application Number : 213535
RX/OTC/DISCN : RX
RLD : Yes
TE Code :
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