SpringWorks Therapeutics Welcomes FDA Approval of GOMEKLI™

SpringWorks Therapeutics Celebrates GOMEKLI™ FDA Approval

GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN.

Approval based on positive data from the ReNeu trial, demonstrating robust results and a manageable safety profile.

SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), known for its dedication to severe rare diseases and cancer treatment, has achieved an important milestone with the FDA's approval of GOMEKLI™ (mirdametinib). This MEK inhibitor is now available for adult and pediatric patients aged 2 years and older suffering from symptomatic plexiform neurofibromas (PN) related to neurofibromatosis type 1 (NF1) that are not suitable for complete surgical removal.

Understanding the Significance of GOMEKLI™

GOMEKLI™ represents hope for a community that has long awaited effective treatment options. According to Saqib Islam, the Chief Executive Officer of SpringWorks, the approval marks a significant step in addressing the unmet needs of NF1-PN patients. He expressed gratitude to all those involved in the clinical trials that contributed to bringing this therapy to fruition.

The approval underscores the clinical need for effective treatments in the NF1 community, which currently affects around 100,000 individuals across the United States. As many as 50% of patients with NF1 may experience plexiform neurofibromas, which can lead to severe disfigurement and impact on quality of life.

Inside the ReNeu Trial Results

The FDA's endorsement was bolstered by positive outcomes from the Phase 2b ReNeu clinical trial that evaluated 114 patients with NF1-PN. The trial revealed impressive results: a confirmed objective response rate (ORR) of 41% in adults and 52% in children, demonstrating the drug's efficacy in reducing tumor volumes significantly.

Patients experienced deep and sustainable improvements in their conditions, with notable reductions in pain and better quality of life reported throughout the trial. A staggering 88% of adults and 90% of children who showed a response maintained their benefits for at least one year, highlighting the treatment's potential longevity in efficacy.

Safe and Effective Treatment

Safety is a focal point for SpringWorks, and GOMEKLI™ has showcased a manageable safety profile. Common adverse events reported during the trials included rash, diarrhea, nausea, and fatigue. With careful monitoring, these side effects were effectively addressed, allowing patients to remain on treatment without significant interruptions.

Additionally, the approval included the granting of a rare pediatric disease priority review voucher, emphasizing the commitment to advancing treatments for pediatric patients.

Looking Ahead

GOMEKLI™ is expected to be available through specialty pharmacies within a short time frame, which will provide immediate access to the treatment. Furthermore, SpringWorks is actively supporting patients through its CareConnections™ program, offering personalized support, insurance information, and educational resources to ease the treatment journey.

In the European markets, SpringWorks’ application for GOMEKLI™ is currently under review, with a decision expected in the near future. This could broaden access to the medicine for a larger patient base beyond the U.S.

About SpringWorks Therapeutics

SpringWorks Therapeutics is committed to improving patient outcomes in severe rare diseases and cancer. With two FDA approvals in just 18 months, the company is making great strides. The launch of GOMEKLI™ is not only a victory for the company but for every patient and family affected by NF1-PN.

SpringWorks is also advancing various novel targeted therapies aimed at addressing severe health conditions that require innovative solutions. For more detailed support, individuals are encouraged to visit the SpringWorks official website for educational material and assistance.

Frequently Asked Questions

What is GOMEKLI™ used for?

GOMEKLI™ is approved for treating symptomatic plexiform neurofibromas in patients with neurofibromatosis type 1, especially those who cannot undergo complete surgical removal.

What are the common side effects of GOMEKLI™?

Common side effects include rash, diarrhea, nausea, musculoskeletal pain, and fatigue.

What is the significance of the FDA's approval?

The FDA approval of GOMEKLI™ is a significant advancement in treatment options for NF1-PN patients, offering a new therapy that can reduce tumor size and improve symptoms.

How is SpringWorks supporting patients undergoing treatment?

The SpringWorks CareConnections™ program provides tailored support services, including financial guidance and educational resources for patients receiving GOMEKLI™.

When will GOMEKLI™ be available to patients?

GOMEKLI™ is anticipated to be available through specialty pharmacies within a couple of weeks following its FDA approval.

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