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1. Arimoclomol Citrate [mi]
2. 368860-21-3
3. Arimoclomol Citrate [usan]
4. Unii-q85ffy6179
5. Q85ffy6179
6. Orphazyme A/s
7. 3-pyridinecarboximidoyl Chloride, N-((2r)-2-hydroxy-3-(1-piperidinyl)propoxy)-, 1-oxide, 2-hydroxy-1,2,3-propanetricarboxylate (1:1)
8. Arimoclomol Citrate (usan)
9. N-[(2r)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl Chloride;2-hydroxypropane-1,2,3-tricarboxylic Acid
10. N-((2r)-2-hydroxy-3-(1-piperidyl)propoxy)pyridine-3-carboximidoyl Chloride, 1-oxide, 2-hydroxy-1,2,3-propanetricarboxylate
11. N-[(2r)-2-hydroxy-3-(1-piperidyl)propoxy]pyridine-3-carboximidoyl Chloride, 1-oxide, 2-hydroxy-1,2,3-propanetricarboxylate
12. Schembl2681096
13. Da-61204
14. Q27287107
15. 855-142-3
16. Arimoclomol Citrate; 3-pyridinecarboximidoyl Chloride, N-[(2r)-2-hydroxy-3-(1-piperidinyl)propoxy]-, 1-oxide, 2-hydroxy-1,2,3-propanetricarboxylate (1:1); 3-pyridinecarboximidoyl Chloride, N-[(2r)-2-hydroxy-3-(1-piperidinyl)propoxy]-, 1-oxide, 2-hydroxy-1,2,3-propanetricarbox
1. Arimoclomol
| Molecular Weight | 505.9 g/mol |
|---|---|
| Molecular Formula | C20H28ClN3O10 |
| Hydrogen Bond Donor Count | 5 |
| Hydrogen Bond Acceptor Count | 12 |
| Rotatable Bond Count | 11 |
| Exact Mass | Da |
| Monoisotopic Mass | Da |
| Topological Polar Surface Area | 203 |
| Heavy Atom Count | 34 |
| Formal Charge | 0 |
| Complexity | 565 |
| Isotope Atom Count | 0 |
| Defined Atom Stereocenter Count | 1 |
| Undefined Atom Stereocenter Count | 0 |
| Defined Bond Stereocenter Count | 0 |
| Undefined Bond Stereocenter Count | 1 |
| Covalently Bonded Unit Count | 2 |
Treatment of amyotrophic lateral sclerosis
Treatment of amyotrophic lateral sclerosis, Treatment of sporadic inclusion body myositis
Treatment of Niemann-Pick disease, type C
Drugs in Development
Details:
Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C
Lead Product(s): Arimoclomol Citrate,Miglustat
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Approved FDFProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: $150.0 million Upfront Cash: Undisclosed
Deal Type: Agreement April 07, 2025
Lead Product(s) : Arimoclomol Citrate,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : $150.0 million
Deal Type : Agreement
Zevra Sells Rare Disease Review Voucher For $150 Million
Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Undisclosed
April 07, 2025
Details:
Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C
Lead Product(s): Arimoclomol Citrate,Miglustat
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Approved FDFProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: $150.0 million Upfront Cash: Undisclosed
Deal Type: Agreement February 27, 2025
Lead Product(s) : Arimoclomol Citrate,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : $150.0 million
Deal Type : Agreement
Zevra to Sell Rare Pediatric Disease Voucher for $150M
Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Undisclosed
February 27, 2025
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 Citrate,Miglustat
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Phase II/ Phase IIIProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable November 21, 2024
Lead Product(s) : Arimoclomol Citrate,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Inapplicable
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 Citrate,Miglustat
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Approved FDFProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable September 20, 2024
Lead Product(s) : Arimoclomol Citrate,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Inapplicable
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 Citrate,Inapplicable
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Phase II/ Phase IIIProduct Type: Miscellaneous
Sponsor: Cantor Fitzgerald
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Public Offering August 08, 2024
Lead Product(s) : Arimoclomol Citrate,Inapplicable
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Cantor Fitzgerald
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.
Product Name : Miplyffa
Product Type : Miscellaneous
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 Citrate,Inapplicable
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Phase II/ Phase IIIProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable August 02, 2024
Lead Product(s) : Arimoclomol Citrate,Inapplicable
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Inapplicable
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 Citrate,Inapplicable
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Phase II/ Phase IIIProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable July 09, 2024
Lead Product(s) : Arimoclomol Citrate,Inapplicable
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Inapplicable
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 Citrate,Inapplicable
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Phase II/ Phase IIIProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable March 04, 2024
Lead Product(s) : Arimoclomol Citrate,Inapplicable
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Inapplicable
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 Citrate,Inapplicable
Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed
Study Phase: Phase II/ Phase IIIProduct Type: Miscellaneous
Sponsor: XOMA
Deal Size: $58.6 million Upfront Cash: $5.0 million
Deal Type: Agreement January 11, 2024
Lead Product(s) : Arimoclomol Citrate,Inapplicable
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.
Product Name : Undisclosed
Product Type : Miscellaneous
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 Citrate,Inapplicable
Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa
Study Phase: Phase II/ Phase IIIProduct Type: Miscellaneous
Sponsor: Undisclosed
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable January 08, 2024
Lead Product(s) : Arimoclomol Citrate,Inapplicable
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Miplyffa
Product Type : Miscellaneous
Upfront Cash : Inapplicable
January 08, 2024
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PharmaCompass offers a list of Arimoclomol Citrate API manufacturers, exporters & distributors, which can be sorted by GMP, USDMF, JDMF, KDMF, CEP (COS), WC, Price,and more, enabling you to easily find the right Arimoclomol Citrate manufacturer or Arimoclomol Citrate supplier for your needs.
Send us enquiries for free, and we will assist you in establishing a direct connection with your preferred Arimoclomol Citrate manufacturer or Arimoclomol Citrate supplier.
PharmaCompass also assists you with knowing the Arimoclomol Citrate API Price utilized in the formulation of products. Arimoclomol Citrate API Price is not always fixed or binding as the Arimoclomol Citrate Price is obtained through a variety of data sources. The Arimoclomol Citrate Price can also vary due to multiple factors, including market conditions, regulatory modifications, or negotiated pricing deals.
A Arimoclomol Citrate manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of Arimoclomol Citrate, including repackagers and relabelers. The FDA regulates Arimoclomol Citrate manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. Arimoclomol Citrate 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 Citrate supplier is an individual or a company that provides Arimoclomol Citrate active pharmaceutical ingredient (API) or Arimoclomol Citrate finished formulations upon request. The Arimoclomol Citrate suppliers may include Arimoclomol Citrate API manufacturers, exporters, distributors and traders.
Arimoclomol Citrate 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 Citrate 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 Citrate GMP manufacturer or Arimoclomol Citrate GMP API supplier for your needs.
A Arimoclomol Citrate CoA (Certificate of Analysis) is a formal document that attests to Arimoclomol Citrate's compliance with Arimoclomol Citrate specifications and serves as a tool for batch-level quality control.
Arimoclomol Citrate CoA mostly includes findings from lab analyses of a specific batch. For each Arimoclomol Citrate 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 Citrate may be tested according to a variety of international standards, such as European Pharmacopoeia (Arimoclomol Citrate EP), Arimoclomol Citrate JP (Japanese Pharmacopeia) and the US Pharmacopoeia (Arimoclomol Citrate USP).
