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
telitacicept and VBP101. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Telitacicept FDA Regulatory Timeline and Events
Telitacicept is a drug developed by Vor Biopharma for the following indication: Sjögren's Disease.
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.
- Drug:
- telitacicept
- Announced Date:
- March 30, 2026
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced the dosing of the first patient in UPSTREAM SjD, a global, randomized, double-blind, placebo-controlled Phase 3 trial evaluating telitacicept in adult patients with active primary Sjögren's disease (SjD), formerly known as Sjögren's syndrome.
AI Summary
Vor Bio announced it has dosed the first patient in UPSTREAM SjD, a global, randomized, double‑blind, placebo‑controlled Phase 3 trial evaluating telitacicept in adults with active primary Sjögren’s disease (formerly Sjögren’s syndrome). The trial will test whether telitacicept can modify the disease course in a condition that is common, often underdiagnosed, and currently treated mainly with symptom‑focused care rather than approved systemic disease‑modifying drugs.
Telitacicept is an investigational recombinant fusion protein that selectively inhibits BLyS (BAFF) and APRIL, two cytokines that support B cell and plasma cell survival; blocking them aims to reduce autoreactive B cells and autoantibody production. Telitacicept is already approved in China for several autoimmune diseases, and Vor Bio is advancing global development programs—including Phase 3 efforts in SjD and generalized myasthenia gravis—to seek approvals in major regions.
Read Announcement- Drug:
- telitacicept
- Announced Date:
- November 8, 2025
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced that the primary endpoint was achieved in Stage A of a Phase 3 clinical study in China evaluating telitacicept in adults with IgA nephropathy (IgAN).
AI Summary
Vor Bio announced that telitacicept met the primary endpoint in Stage A of a Phase 3 study in China for adults with IgA nephropathy (IgAN). In the 39-week trial, telitacicept reduced 24-hour urine protein-to-creatinine ratio (24h‑UPCR) by 58.9% versus an 8.8% reduction with placebo (p<0.0001), showing a rapid and clinically meaningful drop in proteinuria.
The multicenter, randomized, double-blind study enrolled 318 high‑risk patients on stable standard therapy and compared weekly 240 mg telitacicept to placebo. Telitacicept also achieved statistically significant benefits across key secondary endpoints, including stabilization of eGFR and lower risk of ≥30% eGFR decline (6.3% vs. 27.0%). Remission rates (24h‑UPCR thresholds) were much higher with telitacicept than placebo.
Safety was favorable: most adverse events were mild or moderate, and serious adverse events were less frequent with telitacicept (2.5% vs. 8.2%). RemeGen has submitted a BLA to China’s regulator for IgAN based on these results.Read Announcement
- Drug:
- telitacicept
- Announced Date:
- October 29, 2025
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced that its collaborator, RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), presented 48-week open-label extension (OLE) data from its Phase 3 study in China evaluating telitacicept in patients with gMG.
AI Summary
Vor Bio’s collaborator RemeGen presented 48-week open-label extension (OLE) data from its Phase 3 trial in China evaluating telitacicept in gMG at the AANEM Annual Meeting. The OLE followed a 24-week double-blind phase, with placebo patients crossing over to 240 mg telitacicept.
At week 48, patients on telitacicept throughout showed a mean –7.5-point improvement in MG-ADL, while crossover patients achieved –6.3. All continuous patients reached at least a 2-point MG-ADL gain, with 96.2% hitting ≥3-point goals versus 90.2% of crossover participants.
On QMG scores, continuous patients improved by –9.8 points and crossover by –9.3, with ≥5-point reductions in 94.2% and 90.2% of patients, respectively. No new safety signals emerged; injection-site reactions were mild and self-resolving.
These sustained efficacy and safety results support telitacicept’s potential as a best-in-disease therapy in gMG, as RemeGen and Vor Bio advance a global Phase 3 program across 14 countries.
Read Announcement- Drug:
- telitacicept
- Announced Date:
- October 22, 2025
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced that it will host a live webcast to present and discuss the newly disclosed, late-breaking 48-week Phase 3 clinical data in China for telitacicept in primary Sjögren's disease.
AI Summary
Vor Bio announced it will host a live webcast on Tuesday, October 28, 2025 at 4:30 PM Eastern Time to discuss late-breaking, 48-week Phase 3 data from a China study of telitacicept in primary Sjögren’s disease. The trial was conducted by collaborator RemeGen Co., Ltd.
During the webcast, Vor Bio management and Dr. Ronald van Vollenhoven will review key efficacy and safety findings, including performance against end points and side effects.
Listeners can register to listen only, join the live Q&A session, or access a replay posted on the company’s investor website two hours after the webcast. The replay will be available for 30 days. Analysts or investors can sign up using links on the Vor Bio site.
Vor Bio is a clinical-stage biotech company advancing telitacicept, a novel dual-target fusion protein, toward potential commercialization for serious autoimmune diseases worldwide.
Read Announcement- Drug:
- telitacicept
- Announced Date:
- October 17, 2025
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced that clinical data from Stage A of a Phase 3 study in China sponsored by Vor Bio's collaborator RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), which evaluated telitacicept in adults with IgA neuropathy (IgAN), will be presented as a late-breaking oral presentation at American Society of Nephrology's (ASN) Kidney Week 2025 being held Nov 5-9, 2025, in Houston, Texas.
AI Summary
Vor Bio announced that clinical data from Stage A of a Phase 3 study in China, sponsored by its collaborator RemeGen Co., Ltd., will be shared as a late-breaking oral presentation at the American Society of Nephrology’s Kidney Week 2025 in Houston, Texas (Nov 5–9, 2025).
The abstract, titled “Efficacy and Safety of Telitacicept in Patients with IgA Nephropathy: Results from Stage A of a Phase 3 Clinical Study,” will appear in the High-Impact Clinical Trials – 2 session on November 8, 2025 from 10:45–11:00 am CT.
Topline results reported a 55% reduction in 24-hour urine protein-to-creatinine ratio at 39 weeks versus placebo (p<0.0001) and a favorable safety profile. RemeGen has also submitted a Biologics License Application to China’s National Medical Products Administration, which could make telitacicept its fifth approved indication in that market.
Read Announcement- Drug:
- telitacicept
- Announced Date:
- October 16, 2025
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced that results from a Phase 3 study in China evaluating telitacicept in systemic lupus erythematosus (SLE) sponsored by its collaborator, RemeGen Co., Ltd., (HKEX: 9995, SHA: 688331), were published in The New England Journal of Medicine (NEJM).
AI Summary
On Oct. 16, 2025, Vor Bio and its collaborator RemeGen announced that results from a Phase 3 study of telitacicept in Chinese patients with active systemic lupus erythematosus (SLE) were published in The New England Journal of Medicine. This publication underscores the global recognition of clinical research conducted in China.
The randomized trial enrolled 335 patients across 42 hospitals and met its primary endpoint at week 52. A modified SLE Responder Index-4 response was seen in 67.1% of patients treated with telitacicept, versus 32.7% on placebo (P<0.001). Clinical benefits appeared by week 4 and were maintained through one year.
Telitacicept works by dual inhibition of BAFF and APRIL, key proteins that support B-cell survival. This mechanism produced significant and durable reductions in disease activity alongside a favorable safety profile. The results validate a transformative B-cell approach and support telitacicept’s potential as a new standard of care for lupus worldwide.
Read Announcement- Drug:
- telitacicept
- Announced Date:
- October 14, 2025
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced that its collaborator, RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), reported positive 48-week results from its Phase 3 study conducted in China evaluating telitacicept in primary Sjögren's disease.
AI Summary
Vor Bio announced that its collaborator, RemeGen Co., Ltd., reported positive 48-week results from a Phase 3 study in China evaluating telitacicept for primary Sjögren’s disease. The trial met its primary endpoint of change in ESSDAI (EULAR Sjögren’s Syndrome Disease Activity Index) at week 24 and all secondary endpoints, with highly significant p values (p < 0.0001) for both the 160 mg and 80 mg doses versus placebo.
In the 160 mg group, 71.8% of patients achieved at least a 3-point ESSDAI reduction by week 24 compared to 19.3% on placebo. This benefit was maintained through week 48, along with meaningful improvements in patient-reported symptoms (ESSPRI) and a favorable safety profile. Adverse events were mostly mild to moderate, matching what was seen in earlier studies.
Based on these durable and clinically meaningful results, Vor Bio is evaluating the timing of a global Phase 3 clinical study in primary Sjögren’s disease. The full data will be presented in a late-breaking poster session at the American College of Rheumatology Convergence 2025 in Chicago.
Read Announcement- Drug:
- telitacicept
- Announced Date:
- September 29, 2025
- Indication:
- Sjögren's Disease
Announcement
Vor Bio announced that clinical data from the Phase 3 study in China evaluating telitacicept in adults with primary Sjögren's disease, a study sponsored by Vor's collaborator RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), will be presented as a late-breaking poster presentation at ACR Convergence 2025, being held October 24-29, 2025, at McCormick Place in Chicago, Illinois.
AI Summary
Vor Bio announced that clinical data from the Phase 3 study in China evaluating telitacicept in adults with primary Sjögren’s disease will be presented as a late-breaking poster at ACR Convergence 2025. The study was sponsored by Vor’s collaborator RemeGen Co., Ltd. This showcase will take place October 24–29, 2025, at McCormick Place in Chicago, Illinois.
The poster, titled “Efficacy and Safety of Telitacicept in Patients with Sjögren’s Disease: Results from a Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 3 Clinical Study,” is scheduled for Tuesday, October 28 from 10:30 AM to 12:30 PM CT as part of the Late-Breaking Posters session. Telitacicept is a dual-target fusion protein that blocks two key cytokines driving B cell activity and autoantibody production. In early September, RemeGen announced that China’s NMPA accepted its Biologics License Application for telitacicept in primary Sjögren’s disease, which would become its fourth approved indication in China.
Read Announcement
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.
- Drug:
- VBP101
- Announced Date:
- March 20, 2025
- Indication:
- Study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™.
Announcement
Vor Bio provided a business update.
AI Summary
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.
Read Announcement- Drug:
- VBP101
- Announced Date:
- September 6, 2024
- Indication:
- Study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™.
Announcement
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™.
AI Summary
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.
Read Announcement
