Vepdegestrant (ARV-471): FDA Fast Track Designation in ER+/HER2- Metastatic Breast Cancer

Vepdegestrant is an investigational drug belonging to a new class of medications called PROTAC (PROteolysis TArgeting Chimera) protein degraders

Charles H. Weaver, MD
3–4 minutes
Home » Breast Cancer » Vepdegestrant (ARV-471): FDA Fast Track Designation in ER+/HER2- Metastatic Breast Cancer

The FDA recently granted fast track designation to the investigational treatment vepdegestrant (ARV-471) for use in patients with ER-positive/HER2-negative locally advanced or metastatic breast cancer who previously received endocrine-based therapy.

The phase 1/2 VERITAC study evaluated vepdegestrant together with palbociclib (Ibrance) in patients with ER-positive, HER2-negative breast cancer that had spread or could not be removed by surgery.

Key findings:

  1. Effectiveness:
    • The treatment helped slow cancer growth in about 63% of patients.
    • For patients with a specific gene mutation (ESR1), the treatment was even more effective, helping 72% of patients.
    • The treatment shrank tumors in 42% of patients with measurable disease.
  2. How long it worked:
    • On average, the treatment kept cancer from growing for about 11 months.
    • For some patients who hadn’t used similar drugs before, it worked for over 19 months.
  3. Side effects:
    • The most common side effect was low white blood cell count (neutropenia), which all patients experienced to some degree.
    • Other common side effects included fatigue, low platelet count, and anemia.
    • Most side effects were manageable.

This study included patients who had already tried several other treatments, making these results particularly promising. Researchers are now planning larger studies to confirm these findings and potentially make this treatment available to more patients in the future.

Researchers continue to evaluate vepdegestrant in the ongoing Phase 3 VERITAC-2 study and in a Phase 3 trial called the VERITAC-3 trial. The VERITAC-3 study is evaluating vepdegestrant combined with palbociclib (Ibrance) in patients who haven’t had any treatment for advanced breast cancer yet.

Researchers are also looking at combining vepdegestrant with other cancer drugs, including:

    • Abemaciclib (Verzenio)
    • Ribociclib (Kisqali)
    • Samuraciclib
    • Everolimus (Afinitor)
    • A novel CDK4 inhibitor

About Vepdegestrant

Vepdegestrant is an investigational drug being developed to treat certain types of breast cancer. It works in a unique way by targeting and breaking down estrogen receptors in cancer cells. This drug belongs to a new class of medications called PROTAC (PROteolysis TArgeting Chimera) protein degraders. By reducing the number of estrogen receptors, vepdegestrant aims to slow down or stop the growth of estrogen-dependent breast tumors.

About FDA Fast Track Designation

FDA Fast Track designation is a process designed to expedite the development and review of drugs that treat serious conditions and fill an unmet medical need. Key aspects of Fast Track designation include:

  1. Purpose: To facilitate faster development and approval of important new drugs, getting them to patients earlier.
  2. Eligibility: Drugs that treat serious conditions and address an unmet medical need. Serious conditions are generally those affecting survival, day-to-day functioning, or likely to progress to more serious states if left untreated.
  3. Benefits:
    • More frequent meetings and written communication with the FDA
    • Eligibility for Accelerated Approval and Priority Review
    • Rolling Review, allowing submission of completed sections of the drug application for review before the entire application is finished
  4. Application: Drug companies must request Fast Track designation, which can be done at any time during the drug development process.
  5. Review time: The FDA aims to make a decision on Fast Track requests within 60 days.
  6. Impact: Fast Track designation can significantly reduce development timelines and help identify and address potential issues early in the process.

Fast Track is one of several FDA programs designed to accelerate drug development and approval for serious conditions, alongside Breakthrough Therapy, Accelerated Approval, and Priority Review.

More Reading

Treatment of Stage IV – Metastatic Breast Cancer

Endocrine-Hormone Therapy for Advanced-Metastatic Breast Cancer

Endocrine Therapy

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Reference:

Campone M, et al. VERITAC-2: a Phase 3 study of vepdegestrant, a PROteolysis TArgeting Chimera (PROTAC) estrogen receptor (ER) degrader, vs fulvestrant in ER–positive/human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer. Presented at the 2023 San Antonio Breast Cancer Symposium. PO1-19-12.

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