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Inotersen
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Looking for Inotersen API 1492984-65-2?
- Description:
- Here you will find a list of producers, manufacturers and distributors of Inotersen. You can filter on certificates such as GMP, FDA, CEP, Written Confirmation and more. Send inquiries for free and get in direct contact with the supplier of your choice.
- API | Excipient name:
- Inotersen
- Synonyms:
- Cas Number:
- 1492984-65-2
- DrugBank number:
- DB14713
- Unique Ingredient Identifier:
- 0IEO0F56LV
General Description:
Inotersen, identified by CAS number 1492984-65-2, is a notable compound with significant therapeutic applications. Inotersen is a transthyretin-directed antisense oligonucleotide for the treatment of the polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adults. It was FDA approved in October 2018. Inotersen has been shown to improve the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis . Hereditary transthyretin amyloidosis is caused by single-nucleotide variants in the gene encoding transthyretin (TTR), which lead to transthyretin misfolding and the deposition of amyloid substance systemically. Progressive amyloid accumulation may lead to multiorgan dysfunction and death .
Indications:
This drug is primarily indicated for: Inotersen is a transthyretin-directed antisense oligonucleotide indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.
Metabolism:
Inotersen undergoes metabolic processing primarily in: Inotersen is metabolized by nucleases to nucleotides of various lengths. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.
Absorption:
The absorption characteristics of Inotersen are crucial for its therapeutic efficacy: Following subcutaneous administration, inotersen is absorbed rapidly into systemic circulation in a dose-dependent fashion, with the median time to maximum plasma concentrations (Cmax) of 2 to 4 hours. The drug's ability to rapidly penetrate into cells ensures quick onset of action.
Half-life:
The half-life of Inotersen is an important consideration for its dosing schedule: The terminal elimination half-life is 32.3 (29.4, 35.5) days. This determines the duration of action and helps in formulating effective dosing regimens.
Protein Binding:
Inotersen exhibits a strong affinity for binding with plasma proteins: Inotersen is highly bound to human plasma proteins (>94%) and the fraction bound is independent of drug concentration. This property plays a key role in the drug's pharmacokinetics and distribution within the body.
Route of Elimination:
The elimination of Inotersen from the body primarily occurs through: Mainly cleared through metabolism. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.
Volume of Distribution:
Inotersen is distributed throughout the body with a volume of distribution of: Rapidly distributes broadly to tissues, with the highest concentrations observed in the kidney and liver. This drug does not cross the blood-brain barrier. This metric indicates how extensively the drug permeates into body tissues.
Clearance:
The clearance rate of Inotersen is a critical factor in determining its safe and effective dosage: Inotersen is mainly cleared through metabolism, and the total body clearance (mean and 90% confidence interval) is 3.18 (3.08, 3.29) L/h. It reflects the efficiency with which the drug is removed from the systemic circulation.
Pharmacodynamics:
Inotersen exerts its therapeutic effects through: The pharmacodynamic effects of inotersen were studied in hereditary transthyretin-mediated amyloidosis (hATTR) amyloidosis patients treated with 284 mg of inotersen via subcutaneous injection once weekly. With repeated dosing, the mean percentage decreases from baseline in serum TTR (transthyretin) from week 13 to Week 65 of treatment were measured from 68%-74% (median range: 75% to 79%). Similar TTR reductions were seen regardless of TTR mutation, sex, age, or race. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.
Mechanism of Action:
Inotersen functions by: Inotersen is an antisense oligonucleotide that causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.
Toxicity:
Categories:
Inotersen is categorized under the following therapeutic classes: Antisense Elements (Genetics), Antisense Oligonucleotides, Compounds used in a research, industrial, or household setting, Decreased RNA Integrity, DNA, Antisense, Increased Protein Breakdown, Laboratory Chemicals, Molecular Probes, Nephrotoxic agents, Nervous System, Nucleic Acid Probes, Nucleic Acids, Nucleic Acids, Nucleotides, and Nucleosides, Nucleotides, Oligodeoxyribonucleotides, Antisense, Oligonucleotides, Polynucleotides, Potential QTc-Prolonging Agents, QTc Prolonging Agents, Transthyretin-directed RNA Interaction. These classifications highlight the drug's diverse therapeutic applications and its importance in treating various conditions.
Experimental Properties:
Further physical and chemical characteristics of Inotersen include:
- Water Solubility: soluble
- Molecular Weight: 7183.077
- Molecular Formula: C230H318N69O121P19S19
Inotersen is a type of Central Nervous System Agents
Central Nervous System (CNS) Agents are a crucial category of pharmaceutical Active Pharmaceutical Ingredients (APIs) that specifically target the central nervous system. The CNS encompasses the brain and spinal cord, playing a vital role in regulating and controlling various bodily functions, including cognition, movement, emotions, and sensory perception. These agents are designed to interact with specific receptors, enzymes, or ion channels within the CNS to modulate neural activity and restore normal functioning.
CNS agents comprise a diverse range of pharmaceutical APIs, including analgesics, anesthetics, antipsychotics, sedatives, hypnotics, anti-epileptics, and antidepressants. Each subcategory addresses distinct neurological disorders and conditions. For instance, analgesics alleviate pain by targeting receptors in the brain and spinal cord, while antipsychotics are employed to manage psychosis symptoms in mental illnesses such as schizophrenia.
The development of CNS agents involves rigorous research, molecular modeling, and extensive clinical trials to ensure safety, efficacy, and specific target engagement. Pharmaceutical companies invest significant resources in identifying novel drug targets, synthesizing new compounds, and optimizing their pharmacological properties. These agents undergo rigorous regulatory evaluations and must adhere to stringent quality standards and guidelines.
Given the prevalence of CNS disorders globally, the market demand for effective CNS agents is substantial. The development of innovative CNS APIs not only improves patient outcomes but also provides valuable commercial opportunities for pharmaceutical companies. Continued advancements in CNS agent research and development hold the promise of groundbreaking therapies that can improve the quality of life for individuals affected by neurological conditions.
