LUM

Vertex Pharmaceuticals Incorporated · FDA-approved active Small molecule

LUM is a Tankyrase inhibitor / Wnt signaling modulator Small molecule drug developed by Vertex Pharmaceuticals Incorporated. It is currently FDA-approved for Short bowel syndrome (SBS). Also known as: lumacaftor, VX-809.

LUM001 is a β-catenin modulator that enhances Wnt/β-catenin signaling to promote the expansion and function of intestinal stem cells and barrier function.

LUM001 is a β-catenin modulator that enhances Wnt/β-catenin signaling to promote the expansion and function of intestinal stem cells and barrier function. Used for Short bowel syndrome (SBS).

At a glance

Generic name	LUM
Also known as	lumacaftor, VX-809
Sponsor	Vertex Pharmaceuticals Incorporated
Drug class	Tankyrase inhibitor / Wnt signaling modulator
Target	Tankyrase (TNKS1/TNKS2)
Modality	Small molecule
Therapeutic area	Gastroenterology
Phase	FDA-approved

Mechanism of action

LUM001 works by inhibiting tankyrase, which stabilizes β-catenin and activates Wnt signaling in intestinal epithelial cells. This mechanism promotes intestinal stem cell proliferation and differentiation, enhancing intestinal barrier integrity and regeneration. It is designed to treat conditions characterized by intestinal epithelial damage or dysfunction.

Approved indications

• Short bowel syndrome (SBS)

Common side effects

• Diarrhea
• Nausea
• Abdominal pain
• Vomiting

Key clinical trials

• Platform Study to Evaluate the Efficacy and Safety of Anti-malarial Agents in Participants With Uncomplicated Plasmodium Falciparum Malaria (Cohort B2) (PHASE2)
• Investigating the Pharmacology of Tafenoquine in Papua New Guinean Children With Uncomplicated Malaria (PHASE4)
• Feasibility of the LUM Imaging System for Detection of Gastrointestinal Cancers (PHASE1, PHASE2)
• Feasibility of the LUM Imaging System for Peritoneal Surface Malignancies (PHASE1, PHASE2)
• The Remeasure Trial (NA)
• Spinal Stimulation and Mobility Devices (NA)
• Transcripts with Retained H/ACA Box SnoRNA Sequences As Biomarkers for Estrogen Dependence in Lum-B Breast Carcinomas
• Feasibility Study of Intraoperative Detection of Residual Cancer in Breast Cancer Patients (PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• LUM CI brief — competitive landscape report
• LUM updates RSS · CI watch RSS
• Vertex Pharmaceuticals Incorporated portfolio CI

Frequently asked questions about LUM

What is LUM?

LUM is a Tankyrase inhibitor / Wnt signaling modulator drug developed by Vertex Pharmaceuticals Incorporated, indicated for Short bowel syndrome (SBS).

How does LUM work?

LUM001 is a β-catenin modulator that enhances Wnt/β-catenin signaling to promote the expansion and function of intestinal stem cells and barrier function.

What is LUM used for?

LUM is indicated for Short bowel syndrome (SBS).

Who makes LUM?

LUM is developed and marketed by Vertex Pharmaceuticals Incorporated (see full Vertex Pharmaceuticals Incorporated pipeline at /company/vertex-pharmaceuticals-incorporated).

Is LUM also known as anything else?

LUM is also known as lumacaftor, VX-809.

What drug class is LUM in?

LUM belongs to the Tankyrase inhibitor / Wnt signaling modulator class. See all Tankyrase inhibitor / Wnt signaling modulator drugs at /class/tankyrase-inhibitor-wnt-signaling-modulator.

What development phase is LUM in?

LUM is FDA-approved (marketed).

What are the side effects of LUM?

Common side effects of LUM include Diarrhea, Nausea, Abdominal pain, Vomiting.

What does LUM target?

LUM targets Tankyrase (TNKS1/TNKS2) and is a Tankyrase inhibitor / Wnt signaling modulator.

Related

• Drug class: All Tankyrase inhibitor / Wnt signaling modulator drugs
• Target: All drugs targeting Tankyrase (TNKS1/TNKS2)
• Manufacturer: Vertex Pharmaceuticals Incorporated — full pipeline
• Therapeutic area: All drugs in Gastroenterology
• Indication: Drugs for Short bowel syndrome (SBS)
• Also known as: lumacaftor, VX-809