This section highlights FDA-related milestones and regulatory updates for drugs developed by SpringWorks Therapeutics (SWTX).
Over the past two years, SpringWorks Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Mirdametinib and Nirogacestat. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Mirdametinib - FDA Regulatory Timeline and Events
Mirdametinib is a drug developed by SpringWorks Therapeutics for the following indication: Adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN).
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Mirdametinib
- Announced Date:
- May 23, 2025
- Indication:
- Adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN)
Announcement
SpringWorks announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a conditional marketing authorization for mirdametinib, a MEK inhibitor, for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in pediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above.
AI Summary
SpringWorks Therapeutics announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion for mirdametinib. This MEK inhibitor is proposed for treating symptomatic, inoperable plexiform neurofibromas (PN) in both children and adults with neurofibromatosis type 1 (NF1) aged 2 years and older. If approved, mirdametinib would be the first treatment available in the European Union specifically for NF1-PN across all age groups. The medicine is set to be available in 1 mg and 2 mg capsules, as well as a 1 mg dispersible tablet that dissolves in water for easy consumption. The European Commission is expected to review the CHMP’s recommendation and issue a final decision on conditional marketing authorization in the third quarter of 2025.
Read Announcement- Drug:
- Mirdametinib
- Announced Date:
- February 11, 2025
- Indication:
- Adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN)
Announcement
SpringWorks Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has approved GOMEKLI™ (mirdametinib), SpringWorks' MEK inhibitor, for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.1
AI Summary
The U.S. FDA has approved GOMEKLI™ (mirdametinib), the first and only medicine for treating both adults and children aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas that cannot be completely removed by surgery. Developed by SpringWorks Therapeutics, this MEK inhibitor showed robust responses in the Phase 2b ReNeu trial, demonstrating significant tumor shrinkage and durable, deep reductions in tumor volume. The clinical data provided important evidence of early and sustained improvements in pain and quality of life, with a manageable safety profile for both adults and children. Additionally, FDA approval came with a rare pediatric disease priority review voucher, underscoring the potential impact of GOMEKLI on patients facing this debilitating condition.
Read Announcement- Drug:
- Mirdametinib
- Announced Date:
- August 28, 2024
- Indication:
- Adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN)
Announcement
SpringWorks Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) for mirdametinib, an investigational MEK inhibitor, for the treatment of adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).
AI Summary
SpringWorks Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application for mirdametinib. This investigational MEK inhibitor is being developed to treat both adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The FDA granted Priority Review for the application and set a decision target date of February 28, 2025. At this time, the FDA does not plan to convene an advisory committee meeting to discuss the application.
Mirdametinib has the potential to become an important treatment option for patients who currently have limited alternatives. The acceptance of the NDA marks a significant step in SpringWorks’ efforts to address the unmet medical needs of individuals with NF1-PN, offering hope for improved outcomes and quality of life.
Read Announcement- Drug:
- Mirdametinib
- Announced Date:
- August 28, 2024
- Estimated Event Date Range:
- February 28, 2025 - February 28, 2025
- Target Action Date:
- February 28, 2025
- Indication:
- Adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN)
Announcement
SpringWorks Therapeutics, Inc announced that The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2025
AI Summary
SpringWorks Therapeutics recently announced that the FDA has accepted its New Drug Application for mirdametinib, an investigational MEK inhibitor intended for treating neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) in both adults and children. The agency granted the application Priority Review status, which can help speed up the process if the drug shows significant benefits over existing options.
Furthermore, the FDA has set a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2025. This means the agency is aiming to complete its review and provide a decision by that date. This milestone brings hope to patients with NF1-PN who currently have limited treatment options and represents a key step forward in SpringWorks’ mission to deliver transformative therapies.
Read Announcement- Drug:
- Mirdametinib
- Announced Date:
- July 1, 2024
- Indication:
- Adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN)
Announcement
SpringWorks Therapeutics, Inc announced that the Company has completed the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for mirdametinib, an investigational MEK inhibitor, for the treatment of pediatric and adult patients with neurofibromatosis type 1- associated plexiform neurofibromas (NF1-PN).
AI Summary
SpringWorks Therapeutics has completed the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration for mirdametinib. This investigational MEK inhibitor is designed to treat both children and adults with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN), a rare condition that can cause significant health challenges. The NDA is supported by promising data from the pivotal Phase 2b ReNeu trial, which showed that mirdametinib led to significant tumor shrinkage, reduced pain, and improved quality of life for patients. The trial demonstrated deep, lasting responses with a manageable safety profile. SpringWorks’ CEO Saqib Islam expressed optimism about mirdametinib’s potential to become a best-in-class therapy, and the company is looking forward to engaging with the FDA throughout the review process.
Read Announcement
Nirogacestat - FDA Regulatory Timeline and Events
Nirogacestat is a drug developed by SpringWorks Therapeutics for the following indication: Ovarian Granulosa Cell Tumors.
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:
- Nirogacestat
- Announced Date:
- June 20, 2025
- Indication:
- Ovarian Granulosa Cell Tumors
Announcement
SpringWorks Therapeutics, Inc. announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of nirogacestat, an oral gamma secretase inhibitor, as monotherapy for the treatment of adults with progressing desmoid tumors who require systemic treatment.
AI Summary
SpringWorks Therapeutics recently announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion for nirogacestat. This oral gamma secretase inhibitor is being recommended as a monotherapy for adults with progressing desmoid tumors who require systemic treatment. If approved, nirogacestat will be the first treatment with marketing authorization in the EU for desmoid tumors. The European Commission is expected to review this recommendation and make a final decision in the third quarter of 2025. The positive opinion is based on clinical trial results that showed nirogacestat could significantly lower the risk of disease progression, along with improvements in tumor size and patient-reported outcomes. This development offers hope for patients in Europe, who currently face a lack of approved treatment options for this rare, aggressive condition.
Read Announcement- Drug:
- Nirogacestat
- Announced Date:
- June 20, 2025
- Estimated Event Date Range:
- July 1, 2025 - September 30, 2025
- Target Action Date:
- Q3 2025
- Indication:
- Ovarian Granulosa Cell Tumors
Announcement
SpringWorks Therapeutics, Inc announced that Decision from European Commission expected in the third quarter of 2025
AI Summary
SpringWorks Therapeutics announced that the European Commission (EC) is expected to reach a final decision on nirogacestat in the third quarter of 2025. The EC will review a positive recommendation from the EMA’s Committee for Medicinal Products for Human Use, which supports nirogacestat as a treatment for adults with progressing desmoid tumors needing systemic care. If approved, this drug will be the first and only therapy with marketing authorization in the EU for this rare condition.
Recent results from the Phase 3 DeFi trial showed that nirogacestat can improve progression-free survival and overall quality of life. This potential breakthrough gives hope to patients with limited treatment options and highlights the company’s dedication to advancing innovative therapies across Europe.
Read Announcement- Drug:
- Nirogacestat
- Announced Date:
- April 27, 2025
- Estimated Event Date Range:
- April 1, 2025 - June 30, 2025
- Target Action Date:
- Q2 2025
- Indication:
- Ovarian Granulosa Cell Tumors
Announcement
SpringWorks Therapeutics, Inc. that the Company anticipates the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will adopt an opinion on the marketing authorization application (MAA) for nirogacestat, an oral gamma secretase inhibitor, for the treatment of adults with desmoid tumors in the second quarter of 2025.
AI Summary
SpringWorks Therapeutics, Inc. announced that it expects an opinion on its marketing authorization application (MAA) for nirogacestat soon. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) is set to adopt its opinion in the second quarter of 2025. Nirogacestat is an oral gamma secretase inhibitor designed for the treatment of adults with desmoid tumors.
This development is an important step for the company as it works to expand treatment options for patients with this rare condition. The anticipated decision by the CHMP highlights the ongoing efforts by SpringWorks to address severe diseases with its innovative therapies. The outcome could have a significant impact on patients looking for alternative treatment solutions and further strengthens SpringWorks’ commitment to improving lives through its research and development programs.
Read Announcement- Drug:
- Nirogacestat
- Announced Date:
- May 23, 2024
- Indication:
- Ovarian Granulosa Cell Tumors
Announcement
SpringWorks announced that four abstracts from the company's portfolio will be presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting being held May 31 to June 4, 2024.
AI Summary
SpringWorks Therapeutics announced that four abstracts from its portfolio will be presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, scheduled for May 31 to June 4, 2024. One key abstract highlights results from the pivotal Phase 2b ReNeu trial, which evaluated mirdametinib in both adults and children with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The trial, the largest multicenter study in this area, demonstrated notable tumor volume reductions and improvements in pain and quality of life. In addition, additional data from the Phase 3 DeFi trial of nirogacestat in adults with desmoid tumors will be shared in oral and poster sessions, providing further insights into safety and efficacy. These presentations emphasize SpringWorks’ commitment to innovative therapies for severe rare diseases and cancer, and they will showcase important clinical advancements at the meeting.
Read Announcement- Drug:
- Nirogacestat
- Announced Date:
- April 24, 2024
- Indication:
- Ovarian Granulosa Cell Tumors
Announcement
SpringWorks Therapeutics announced that data from the Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor, in pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) will be presented in an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, being held May 31 to June 4, 2024. SpringWorks recently initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for mirdametinib in pediatric and adult patients with NF1-PN.
AI Summary
SpringWorks Therapeutics announced that data from its Phase 2b ReNeu trial evaluating mirdametinib—a promising investigational MEK inhibitor—will be featured in an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting held from May 31 to June 4. The study involves pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN), a condition that causes severe tumor-related complications. The presented data, which include assessments of tumor volume reduction verified by MRI and independent review, represent a key element of the company’s New Drug Application (NDA) submission to the U.S. Food and Drug Administration. This submission is part of a rolling process aimed at providing a new treatment option for patients suffering from NF1-PN, addressing a significant unmet need in both children and adults affected by this rare genetic disorder.
Read Announcement
