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1. Tofersen
| Molecular Weight | 7128 g/mol |
|---|---|
| Molecular Formula | C230H317N72O123P19S15 |
| XLogP3 | -16.7 |
| Hydrogen Bond Donor Count | 44 |
| Hydrogen Bond Acceptor Count | 168 |
| Rotatable Bond Count | 156 |
| Exact Mass | Da |
| Monoisotopic Mass | Da |
| Topological Polar Surface Area | 2920 |
| Heavy Atom Count | 459 |
| Formal Charge | 0 |
| Complexity | 19900 |
| Isotope Atom Count | 0 |
| Defined Atom Stereocenter Count | 70 |
| Undefined Atom Stereocenter Count | 0 |
| Defined Bond Stereocenter Count | 0 |
| Undefined Bond Stereocenter Count | 0 |
| Covalently Bonded Unit Count | 1 |
Tofersen is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on the reduction in plasma neurofilament light chain (NfL) observed in patients treated with tofersen. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials.
Indications
N - Nervous system
N07 - Other nervous system drugs
N07X - Other nervous system drugs
N07XX - Other nervous system drugs
N07XX22 - Tofersen
ATCvet Code
QN - Nervous system
QN07 - Other nervous system drugs
QN07X - Other nervous system drugs
QN07XX - Other nervous system drugs
QN07XX22 - Tofersen
Absorption
Intrathecal administration of tofersen into the CSF allows tofersen to be distributed from the CSF to central nervous system tissues. The maximum CSF trough concentration occurred at the third dose, which was the last dose of the loading period. There was little to no accumulation for CSF tofersen with monthly dosing after the loading phase. Tofersen is transferred from CSF into the systemic circulation, with a median time to maximum concentration (Tmax) plasma values ranging from 2 to 6 hours. There was no accumulation in plasma tofersen exposure following monthly maintenance dosing.
Route of Elimination
The primary route of elimination has not been characterized.
Tofersen is expected to be metabolized through exonuclease (3'- and 5')-mediated hydrolysis and is not a substrate for, or inhibitor, or inducer of CYP450 enzymes.
The effective half-life in CSF is estimated to be 4 weeks.
Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.
Drugs in Development
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PharmaCompass offers a list of Tofersen API manufacturers, exporters & distributors, which can be sorted by GMP, USDMF, JDMF, KDMF, CEP (COS), WC, and more, enabling you to easily find the right Tofersen manufacturer or Tofersen supplier for your needs.
Send us enquiries for free, and we will assist you in establishing a direct connection with your preferred Tofersen manufacturer or Tofersen supplier.
A Tofersen manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of Tofersen, including repackagers and relabelers. The FDA regulates Tofersen manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. Tofersen 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 Tofersen supplier is an individual or a company that provides Tofersen active pharmaceutical ingredient (API) or Tofersen finished formulations upon request. The Tofersen suppliers may include Tofersen API manufacturers, exporters, distributors and traders.
Tofersen 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 Tofersen GMP manufacturers, exporters & distributors, which can be sorted by USDMF, JDMF, KDMF, CEP (COS), WC, and more, enabling you to easily find the right Tofersen GMP manufacturer or Tofersen GMP API supplier for your needs.
A Tofersen CoA (Certificate of Analysis) is a formal document that attests to Tofersen's compliance with Tofersen specifications and serves as a tool for batch-level quality control.
Tofersen CoA mostly includes findings from lab analyses of a specific batch. For each Tofersen 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.
Tofersen may be tested according to a variety of international standards, such as European Pharmacopoeia (Tofersen EP), Tofersen JP (Japanese Pharmacopeia) and the US Pharmacopoeia (Tofersen USP).
