Overview
Sonlicromanol is under investigation in clinical trial NCT04165239 (The KHENERGYZE Study).
Indication
No indication information available.
Associated Conditions
No associated conditions information available.
Research Report
Sonlicromanol (DB16333): A Comprehensive Clinical and Scientific Review
1. Sonlicromanol: An Overview
1.1. Introduction to Sonlicromanol (KH176)
Sonlicromanol, also identified by its development code KH176, is an orally administered small molecule drug candidate currently under investigation.[1] It is being developed as a potentially first-in-class, disease-modifying therapy for primary mitochondrial diseases (PMD), a group of rare and often devastating conditions with limited treatment options.[3] Sonlicromanol is considered one of the most clinically advanced drug candidates for PMD currently in development.[3]
The primary developmental focus for Sonlicromanol is on patients with PMD arising from the m.3243A>G mutation in the mitochondrial MT-TL1 gene. This specific genetic variation is the most common cause of PMD and is associated with a spectrum of clinical presentations, including Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS), Maternally Inherited Diabetes and Deafness (MIDD), mixed phenotypes (MP), and Chronic Progressive External Ophthalmoplegia (CPEO).[1] This targeted approach towards a genetically defined subgroup, while addressing the most prevalent form of PMD, is strategically significant. Focusing on a specific mutation allows for a more homogeneous patient population in clinical trials, which can be particularly advantageous in the context of rare diseases where patient numbers are inherently small. Such precision may reduce variability in patient responses and enhance the statistical power of studies to detect a therapeutic effect, aligning with the principles of personalized medicine.
1.2. Chemical Properties and Formulation
Sonlicromanol is classified as a small molecule therapeutic. Its fundamental chemical and physical properties are summarized below.
Table 1: Sonlicromanol - Key Identifiers and Properties
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
---|---|---|---|---|---|
2024/06/11 | Phase 3 | Not yet recruiting | |||
2021/04/15 | Phase 2 | Suspended | |||
2019/11/15 | Phase 2 | Completed | |||
2016/09/21 | Phase 2 | Completed | |||
2015/09/09 | Phase 1 | Completed |
FDA Drug Approvals
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No FDA approvals found for this drug. |
EMA Drug Approvals
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No EMA approvals found for this drug. |
HSA Drug Approvals
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No HSA approvals found for this drug. |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
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No NMPA approvals found for this drug. |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
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No PPB approvals found for this drug. |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
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No TGA approvals found for this drug. |
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
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No Health Canada approvals found for this drug. |
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
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No CIMA AEMPS (Spain) approvals found for this drug. |
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
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No Philippines FDA approvals found for this drug. |
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
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No Saudi SFDA approvals found for this drug. |
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
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No Malaysia NPRA approvals found for this drug. |
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
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No UK EMC drug information found for this drug. |
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