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1. Arimoclomol
2. Brx-220
3. Brx220
1. Arimoclomol
2. 289893-25-0
3. Arimoclomol (usan)
4. Brx-220 Free Base
5. Dsstox_cid_31490
6. Dsstox_rid_97375
7. Dsstox_gsid_57701
8. Chembl2107726
9. Dtxsid5057701
10. Schembl12254225
11. Tox21_113718
12. Ncgc00253575-01
13. Hy-106443
14. Cas-289893-25-0
15. Cs-0025833
16. D11374
17. (3z)-n-[(2r)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl Chloride
Molecular Weight | 313.78 g/mol |
---|---|
Molecular Formula | C14H20ClN3O3 |
XLogP3 | 1.2 |
Hydrogen Bond Donor Count | 1 |
Hydrogen Bond Acceptor Count | 5 |
Rotatable Bond Count | 6 |
Exact Mass | 313.1193192 g/mol |
Monoisotopic Mass | 313.1193192 g/mol |
Topological Polar Surface Area | 70.5 Ų |
Heavy Atom Count | 21 |
Formal Charge | 0 |
Complexity | 337 |
Isotope Atom Count | 0 |
Defined Atom Stereocenter Count | 1 |
Undefined Atom Stereocenter Count | 0 |
Defined Bond Stereocenter Count | 1 |
Undefined Bond Stereocenter Count | 0 |
Covalently Bonded Unit Count | 1 |
N - Nervous system
N07 - Other nervous system drugs
N07X - Other nervous system drugs
N07XX - Other nervous system drugs
N07XX17 - Arimoclomol
Details:
Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 21, 2024
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Zevra Launches MIPLYFFA™ in US for Niemann-Pick Disease Type C Treatment
Details : Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.
Brand Name : Miplyffa
Molecule Type : Small molecule
Upfront Cash : Not Applicable
November 21, 2024
Details:
Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: ApprovedProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 20, 2024
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
FDA Approves First Treatment for Niemann-Pick Disease, Type C
Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.
Brand Name : Miplyffa
Molecule Type : Small molecule
Upfront Cash : Not Applicable
September 20, 2024
Details:
The net proceeds from this offering will used to support the pre-commercial launch activities for BRX-345 (arimoclomol) for the treatment of Niemann-Pick Disease Type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: Cantor
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Public Offering August 08, 2024
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Cantor
Deal Size : Undisclosed
Deal Type : Public Offering
Zevra Therapeutics Announces Proposed Public Offering of Common Stock
Details : The net proceeds from this offering will used to support the pre-commercial launch activities for BRX-345 (arimoclomol) for the treatment of Niemann-Pick Disease Type C.
Brand Name : BRX-345
Molecule Type : Small molecule
Upfront Cash : Undisclosed
August 08, 2024
Details:
BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 02, 2024
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
FDA Advisory Committee Supports Arimoclomol as Effective for Niemann-Pick Disease Type C
Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.
Brand Name : BRX-345
Molecule Type : Small molecule
Upfront Cash : Not Applicable
August 02, 2024
Details:
BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 09, 2024
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Zevra Announces FDA Advisory Committee Meeting for Arimoclomol in Niemann-Pick Disease
Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.
Brand Name : BRX-345
Molecule Type : Small molecule
Upfront Cash : Not Applicable
July 09, 2024
Details:
BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 04, 2024
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Zevra Therapeutics Provides FDA Update on PDUFA Action Date for Arimoclomol Treatment
Details : BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.
Brand Name : BRX-345
Molecule Type : Small molecule
Upfront Cash : Not Applicable
March 04, 2024
Details:
XOMA acquired royalty and milestone rights for LadRx Corporation’s BRX-345 (arimoclomol) for Niemann-Pick Disease Type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: XOMA
Deal Size: $58.6 million Upfront Cash: $5.0 million
Deal Type: Agreement January 11, 2024
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : XOMA
Deal Size : $58.6 million
Deal Type : Agreement
FDA Accepts Zevra's Arimoclomol NDA for NPC Type C, Triggering Milestone Payment
Details : XOMA acquired royalty and milestone rights for LadRx Corporation’s BRX-345 (arimoclomol) for Niemann-Pick Disease Type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.
Brand Name : BRX-345
Molecule Type : Small molecule
Upfront Cash : $5.0 million
January 11, 2024
Details:
Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 08, 2024
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Zevra Therapeutics Receives FDA Acceptance of NDA Resubmission for Arimoclomol Treatment
Details : Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.
Brand Name : Undisclosed
Molecule Type : Small molecule
Upfront Cash : Not Applicable
January 08, 2024
Details:
Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed
Study Phase: Phase II/ Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 27, 2023
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.
Brand Name : Undisclosed
Molecule Type : Small molecule
Upfront Cash : Not Applicable
December 27, 2023
Details:
Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. It is being developed as a treatment for Niemann-Pick disease type C (NPC).
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed
Study Phase: Phase IIIProduct Type: Small molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 23, 2023
Lead Product(s) : Arimoclomol
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. It is being developed as a treatment for Niemann-Pick disease type C (NPC).
Brand Name : Undisclosed
Molecule Type : Small molecule
Upfront Cash : Not Applicable
February 23, 2023
https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-sept-2024-bms-wins-landmark-fda-approval-for-schizophrenia-med-sanofi-s-dupixent-okayed-for-copd
ABOUT THIS PAGE
A Arimoclomol manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of Arimoclomol, including repackagers and relabelers. The FDA regulates Arimoclomol manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. Arimoclomol API Manufacturers are required to adhere to Good Manufacturing Practices (GMP) to ensure that their products are consistently manufactured to meet established quality criteria.
A Arimoclomol supplier is an individual or a company that provides Arimoclomol active pharmaceutical ingredient (API) or Arimoclomol finished formulations upon request. The Arimoclomol suppliers may include Arimoclomol API manufacturers, exporters, distributors and traders.
Arimoclomol Active pharmaceutical ingredient (API) is produced in GMP-certified manufacturing facility.
GMP stands for Good Manufacturing Practices, which is a system used in the pharmaceutical industry to make sure that goods are regularly produced and monitored in accordance with quality standards. The FDA’s current Good Manufacturing Practices requirements are referred to as cGMP or current GMP which indicates that the company follows the most recent GMP specifications. The World Health Organization (WHO) has its own set of GMP guidelines, called the WHO GMP. Different countries can also set their own guidelines for GMP like China (Chinese GMP) or the EU (EU GMP).
PharmaCompass offers a list of Arimoclomol GMP manufacturers, exporters & distributors, which can be sorted by USDMF, JDMF, KDMF, CEP (COS), WC, API price, and more, enabling you to easily find the right Arimoclomol GMP manufacturer or Arimoclomol GMP API supplier for your needs.
A Arimoclomol CoA (Certificate of Analysis) is a formal document that attests to Arimoclomol's compliance with Arimoclomol specifications and serves as a tool for batch-level quality control.
Arimoclomol CoA mostly includes findings from lab analyses of a specific batch. For each Arimoclomol CoA document that a company creates, the USFDA specifies specific requirements, such as supplier information, material identification, transportation data, evidence of conformity and signature data.
Arimoclomol may be tested according to a variety of international standards, such as European Pharmacopoeia (Arimoclomol EP), Arimoclomol JP (Japanese Pharmacopeia) and the US Pharmacopoeia (Arimoclomol USP).
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