Overview

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Research Report

Published: May 12, 2025

VX-407: An Investigational Small Molecule Corrector for Autosomal Dominant Polycystic Kidney Disease

I. Introduction to VX-407

A. Overview of VX-407 as an Investigational Therapeutic Agent

VX-407 is an investigational, first-in-class, orally administered small molecule therapeutic agent currently under development.[1] Its primary distinction lies in its targeted mechanism as a "corrector" molecule, designed to address the underlying genetic cause of Autosomal Dominant Polycystic Kidney Disease (ADPKD) in a specific subpopulation of patients.[2] The development of VX-407 represents a strategic initiative by its developer to apply expertise in creating transformative medicines for serious genetic conditions to the field of inherited kidney diseases. Notably, ADPKD marks the tenth distinct disease area to be incorporated into the clinical pipeline of Vertex Pharmaceuticals, underscoring a commitment to broadening its therapeutic reach.[2]

B. Developer: Vertex Pharmaceuticals

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Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants	2025/09/08	NCT07161037	Not Applicable	Recruiting	Vertex Pharmaceuticals Incorporated
Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants	2025/07/20	NCT07074327	Not Applicable	Recruiting	Vertex Pharmaceuticals Incorporated
A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants	2025/06/15	NCT07022119	Phase 1	Recruiting	Vertex Pharmaceuticals Incorporated
A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants	2024/04/03	NCT06345755	Phase 1	Completed	Vertex Pharmaceuticals Incorporated

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
No FDA approvals found for this drug.

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
No EMA approvals found for this drug.

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
No Health Canada approvals found for this drug.

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
No CIMA AEMPS (Spain) approvals found for this drug.

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

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