This section highlights FDA-related milestones and regulatory updates for drugs developed by Verastem (VSTM).
Over the past two years, Verastem has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Avutometinib and RAMP. 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.
Avutometinib FDA Regulatory Timeline and Events
Avutometinib is a drug developed by Verastem for the following indication: For the treatment of low-grade serous ovarian cancer (LGSOC).
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:
- Avutometinib
- Announced Date:
- May 22, 2025
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced positive updated safety and efficacy results from the RAMP 205 Phase 1/2 trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the front-line for patients with metastatic pancreatic ductal adenocarcinoma (PDAC).
AI Summary
Verastem Oncology announced positive updated safety and efficacy results from the RAMP 205 Phase 1/2 trial focused on front-line treatment of metastatic pancreatic ductal adenocarcinoma (PDAC). In the trial’s selected recommended Phase 2 dose, dose level 1, 83% of patients (10 out of 12) achieved a partial response when treated with a combination of avutometinib, defactinib, gemcitabine, and Nab-paclitaxel. These promising outcomes demonstrate strong tumor reduction and a manageable safety profile with no new safety signals.
Based on the encouraging data, plans are underway to expand the trial and begin a registrational Phase 3 study in 2026. These results support the potential of this combination therapy as a novel treatment option for front-line metastatic PDAC, offering hope for improved outcomes in this challenging patient population.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- March 20, 2025
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology highlighted recent progress.
AI Summary
Verastem Oncology recently highlighted significant progress in their clinical pipeline. Their novel combination of avutometinib plus defactinib for KRAS mutant recurrent low‐grade serous ovarian cancer received high praise from the FDA. The agency accepted the New Drug Application under the accelerated approval pathway and granted Priority Review, with a PDUFA action date set for June 30, 2025. CEO Dan Paterson stated that 2025 is expected to be transformational, as the company aims to launch the first FDA-approved treatment for this specific patient group and transition into a fully integrated commercial-stage organization.
Additionally, Verastem is advancing further studies, including initiating a Phase 1/2a trial for VS-7375—an oral KRAS G12D inhibitor—and progressing studies in pancreatic and non-small cell lung cancers. These efforts underline the company’s commitment to expanding treatment options in oncology.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- December 30, 2024
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) under the accelerated approval pathway for avutometinib, an oral RAF/MEK clamp, in combination with defactinib, an oral FAK inhibitor, for the treatment of adult patients with recurrent low-grade serous ovarian cancer (LGSOC), who received at least one prior systemic therapy and have a KRAS mutation.
AI Summary
Verastem Oncology announced that the FDA has accepted its New Drug Application (NDA) for review under the accelerated approval pathway. The application is for a new treatment that combines avutometinib, an oral RAF/MEK clamp, with defactinib, an oral FAK inhibitor. This treatment is aimed at adult patients with recurrent low-grade serous ovarian cancer (LGSOC) who have received at least one prior systemic therapy and have a KRAS mutation.
If approved, this would be the first FDA-approved treatment specifically for this patient group. The NDA, submitted in October 2024, has received Priority Review, with a target action date set for June 30, 2025. Verastem is hopeful that this new therapy will address a significant unmet need and offer a promising new option for patients facing this rare and challenging form of ovarian cancer.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- December 18, 2024
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced preliminary clinical data for the triplet combination of avutometinib and sotorasib plus defactinib in the RAMP 203 Phase 1/2 study in KRAS G12C mutant advanced non-small cell lung cancer (NSCLC).
AI Summary
Verastem Oncology announced early clinical data from the RAMP 203 Phase 1/2 study, which is testing a new triplet combination of avutometinib, sotorasib, and defactinib in patients with advanced non-small cell lung cancer bearing the KRAS G12C mutation. In this trial, no dose-limiting toxicities were observed among patients who had already progressed on earlier treatments. Notably, two of the three patients treated in this cohort experienced initial tumor reductions of at least 20%, and all remain on the study treatment. The addition of defactinib, a focal adhesion kinase (FAK) inhibitor, is designed to boost anti-tumor activity by addressing resistance mechanisms. Verastem plans to enroll more patients and will present further data at a medical meeting in the second half of 2025, highlighting the promising nature of this combination approach in addressing difficult-to-treat lung cancer.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- July 29, 2024
- Estimated Event Date Range:
- January 1, 2025 - March 31, 2025
- Target Action Date:
- Q1 2025
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced that Expect to report updated data from the ongoing RAMP 205 trial in first-line metastatic pancreatic cancer in Q1 2025
AI Summary
Verastem Oncology announced that updated data from its ongoing RAMP 205 trial in first-line metastatic pancreatic cancer is expected in the first quarter of 2025. The RAMP 205 trial is a Phase 1b/2a study evaluating the combination of avutometinib and defactinib with standard chemotherapy. Early results have shown positive trends, and the upcoming report will include data from various dose groups to further assess the safety and potential effectiveness of this novel treatment in a challenging cancer type.
This trial is part of Verastem’s ongoing program aimed at finding new treatment options for patients with metastatic pancreatic cancer, a disease known for its poor outcomes. The forthcoming data update will provide more insights into the combination therapy’s ability to improve outcomes for patients with this aggressive form of cancer.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- July 29, 2024
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to avutometinib, a RAF/MEK clamp, in combination with defactinib, a selective FAK inhibitor, for the treatment of pancreatic cancer.
AI Summary
Verastem Oncology announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to avutometinib, a RAF/MEK clamp, when used in combination with defactinib, a selective FAK inhibitor, for the treatment of pancreatic cancer. This designation highlights the urgent need for new therapies in a disease with very limited treatment options and poor survival outcomes. The combination is designed to offer a novel approach by targeting both RAF/MEK and FAK pathways, potentially leading to a more effective blockade of tumor growth. The treatment is currently being evaluated in the RAMP 205 trial, which is focused on patients with first-line metastatic pancreatic cancer. Verastem plans to release further trial updates in the first quarter of 2025, marking an important step forward in addressing the challenges of treating pancreatic cancer.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- May 24, 2024
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced that it has initiated the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of the combination of avutometinib, a RAF/MEK clamp, and defactinib, a selective FAK inhibitor, for adult patients with recurrent KRAS mutant (KRAS mt) low-grade serous ovarian cancer (LGSOC), who received at least one prior systemic therapy.
AI Summary
Verastem Oncology has initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for accelerated approval of its combination therapy using avutometinib, a RAF/MEK clamp, and defactinib, a selective FAK inhibitor. The NDA targets adult patients with recurrent KRAS mutant (KRAS mt) low‐grade serous ovarian cancer (LGSOC) who have received at least one prior systemic therapy.
The rolling review approach allows the FDA to assess sections of the application as they are completed, starting with the nonclinical and quality modules. The company plans to include a primary efficacy analysis from the RAMP 201 trial with 12 months of follow-up data and expects to complete the full submission in the second half of 2024. The robust and durable responses seen in the trial support the potential of this combination to fill a significant unmet medical need in LGSOC treatment.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- May 23, 2024
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced the initial interim safety and efficacy results from the ongoing RAMP 205 Phase 1/2 clinical trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the first-line in patients with metastatic pancreatic cancer.
AI Summary
Verastem Oncology announced initial interim safety and efficacy results from the ongoing RAMP 205 Phase 1/2 trial, which is evaluating a combination of avutometinib and defactinib with gemcitabine and Nab-paclitaxel as first-line treatment for metastatic pancreatic cancer. In the most mature dose level (cohort 1), 83% of patients (5 out of 6) achieved a confirmed partial response. Although one dose-limiting toxicity was observed, the dose level was later cleared after enrolling additional patients. Researchers are continuing to assess additional dose and schedule cohorts to determine the recommended Phase 2 dose for further study expansion. These encouraging results highlight the potential benefit of targeting the RAS/MAPK pathway, as more than 90% of pancreatic tumors have a KRAS mutation. The trial findings will be presented at the upcoming ASCO Annual Meeting, reflecting Verastem’s commitment to advancing new treatment options for pancreatic cancer patients.
Read Announcement
RAMP 201 FDA Regulatory Timeline and Events
RAMP 201 is a drug developed by Verastem for the following indication: Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
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:
- RAMP 201
- Announced Date:
- July 11, 2025
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced that the primary analysis of the Phase 2 RAMP 201 clinical trial was published online in the Journal of Clinical Oncology (JCO).
AI Summary
Verastem Oncology announced that the primary analysis of its Phase 2 RAMP 201 clinical trial was published online in the Journal of Clinical Oncology (JCO). The study examined the use of the drug combination avutometinib plus defactinib in patients with recurrent low‐grade serous ovarian cancer who had received multiple previous treatments.
The analysis revealed an overall confirmed response rate of 31%, with a higher response in patients with KRAS mutations (44%) compared to those with KRAS wild-type (17%). In addition, 82% of patients had a reduction in target lesions. The median progression-free survival was 12.9 months overall, with improved outcomes in the KRAS-mutant group. These promising results support the potential of this combination therapy to offer a durable and meaningful benefit for patients facing this challenging cancer, while ongoing trials continue to evaluate its effectiveness further.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- May 20, 2025
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Tempus Announces a Collaboration with Verastem to Develop CDx for First-Ever FDA-Approved KRAS-Mutant Recurrent Low-Grade Serous Ovarian Cancer Combination Treatment
AI Summary
Tempus AI and Verastem Oncology have joined forces to develop a companion diagnostic (CDx) test for a new targeted treatment. The collaboration aims to support the first-ever FDA-approved combination treatment with avutometinib and defactinib for KRAS-mutant recurrent low-grade serous ovarian cancer (LGSOC), a rare cancer that often affects younger women and has few treatment options. Tempus has already provided confirmatory testing in Verastem’s Phase 2 RAMP-201 trial using its FDA-approved xT CDx assay. This assay is now being used in the global Phase 3 RAMP-301 trial to help identify patients with KRAS mutations, ensuring they receive the most suitable treatment. The new diagnostic test is a key step to better group patients based on their cancer’s genetic profile, potentially leading to more effective and personalized therapies for LGSOC patients.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- February 20, 2025
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced multiple oral and poster presentations, including an oral presentation of additional analyses from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) trial evaluating the investigational combination of avutometinib plus defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC), at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting on Women's Cancer, to be held on March 14-17 in Seattle, Washington.
AI Summary
Verastem Oncology announced that it will present new results from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) trial at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting on Women’s Cancer in Seattle, Washington, from March 14-17. The oral presentation will reveal additional analyses, including subgroup data by KRAS mutational status, from the study evaluating the combination of avutometinib and defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). This data builds on the primary analysis that supported their New Drug Application currently under Priority Review by the FDA. The presentation is part of Verastem’s ongoing efforts to expand treatment options for patients with RAS/MAPK pathway-driven cancers, and it offers an opportunity for oncologists to gain further insights into the potential benefits of this targeted therapy combination.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- October 9, 2024
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced further details for its late-breaking presentation of mature data from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) clinical trial to be presented as an oral presentation at a plenary session at the International Gynecologic Cancer Society (IGCS) Annual Global Meeting taking place October 16-18, 2024 in Dublin, Ireland.
AI Summary
Verastem Oncology announced details of its late-breaking presentation at the upcoming International Gynecologic Cancer Society (IGCS) Annual Global Meeting, which will be held in Dublin, Ireland from October 16-18, 2024. During the meeting’s plenary session on October 17, 2024 at 11:42 AM ET, the company will showcase mature data from the ongoing Phase 2 RAMP 201 trial (ENGOT-ov60/GOG-3052).
The oral presentation will share updated safety and efficacy findings for the combination of avutometinib, a novel RAF/MEK clamp, and defactinib, an oral selective FAK inhibitor, in patients with recurrent low-grade serous ovarian cancer (LGSOC). Professor Susana N. Banerjee, the global lead investigator, is set to present the data, highlighting metrics such as overall response rate, progression-free survival, and duration of response.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- September 10, 2024
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced the acceptance of a late-breaking abstract of the mature data from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) clinical trial to be presented as an oral presentation at a plenary session at the International Gynecologic Cancer Society (IGCS) Annual Meeting taking place October 16-18, 2024 in Dublin, Ireland.
AI Summary
Verastem Oncology announced that a late-breaking abstract of mature data from its ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) clinical trial has been accepted for an oral presentation. The presentation is scheduled for a plenary session at the International Gynecologic Cancer Society (IGCS) Annual Meeting in Dublin, Ireland, from October 16 to 18, 2024. This abstract will present updated safety and efficacy results for the combination of avutometinib—a RAF/MEK clamp—and defactinib, a selective FAK inhibitor, in patients with low‐grade serous ovarian cancer. It will highlight key outcomes such as overall response rate, progression-free survival, and duration of response, offering important insights into the potential impact of this novel therapeutic approach.
The session at IGCS will provide a platform for experts in the field to engage with the latest findings and further discussion on advancements in ovarian cancer treatment.
Read Announcement
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