Vor Biopharma (VOR) FDA Events

Vor Biopharma's Drug in the FDA Approval Process

This section highlights FDA-related milestones and regulatory updates for drugs developed by Vor Biopharma (VOR). Over the past two years, Vor Biopharma has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as VBP101. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.

VBP101 FDA Regulatory Events

VBP101 is a drug developed by Vor Biopharma for the following indication: Study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Provided Update - March 20,2025

• Drug: VBP101
• Announced Date: March 20, 2025
• Indication: Study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™.

Vor Bio provided a business update.

Vor Bio provided a business update highlighting progress in its cell and genome engineering programs. The company shared that the next clinical data update for its VCAR33 trial is expected in the first half of 2025. This update is part of their ongoing efforts to develop therapies for patients with blood cancers. The VCAR33 trial is a Phase 1/2 study that involves a novel anti-CD33 CAR-T cell therapy, aimed at helping patients with relapsed AML after standard treatments or trem-cel transplants. The update comes as the company continues to strengthen its financial position following a $55.6 million private placement led by Reid Hoffman and RA Capital Management. This enhanced financial backing will support upcoming clinical trials, including the anticipated Phase 1 trial for the combined trem-cel+VCAR33 Treatment System scheduled for the second half of 2025.

New Data - September 6,2024

Phase 1/2

• Drug: VBP101
• Announced Date: September 6, 2024
• Indication: Study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™.

Vor Bio announced new clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™.

Vor Bio recently shared encouraging new clinical data from its ongoing Phase 1/2 VBP101 study in patients with relapsed/refractory AML. In this study, patients received the cell therapy trem‐cel followed by the chemotherapy drug Mylotarg™, and the results were promising. The data showed that all patients achieved reliable engraftment, with primary neutrophil recovery in a median of 9 days and robust platelet recovery by around 16.5 days. Additionally, the treatment provided shielding from Mylotarg’s on-target toxicity, which allowed for a broadened therapeutic window. Early signs of patient benefit were observed, including improved relapse-free survival compared to historical high-risk AML outcomes. These findings support the potential of combining trem-cel with Mylotarg to better manage AML and justify discussions with the FDA to design a pivotal trial.

Vor Biopharma FDA Events - Frequently Asked Questions

Has Vor Biopharma received FDA approval?

As of now, Vor Biopharma (VOR) has not received any FDA approvals for its therapy in the last two years.

What drugs has Vor Biopharma submitted to the FDA?

In the past two years, Vor Biopharma (VOR) has reported FDA regulatory activity for VBP101.

What is the most recent FDA event for Vor Biopharma?

The most recent FDA-related event for Vor Biopharma occurred on March 20, 2025, involving VBP101. The update was categorized as "Provided Update," with the company reporting: "Vor Bio provided a business update."

What conditions do Vor Biopharma's current drugs treat?

Currently, Vor Biopharma has one therapy (VBP101) targeting the following condition: Study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™..