In a groundbreaking development for patients with progressing desmoid tumors, the Food and Drug Administration (FDA) has granted approval for nirogacestat, a novel treatment provided by SpringWorks Therapeutics, Inc. This approval marks a significant milestone as the first-ever sanctioned therapy for desmoid tumors, offering hope and a new avenue of treatment for affected individuals. Let’s delve into the details of this approval and what it means for patients, their families, and healthcare providers.
Understanding Desmoid Tumors
Desmoid tumors are rare, noncancerous growths that can arise in any connective tissue. While not classified as cancer, they can be locally aggressive and challenging to manage. Patients facing progressing desmoid tumors that are not amenable to surgery have been without an approved systemic treatment until now.
Nirogacestat Approval and Trial Overview
The FDA’s approval of nirogacestat comes after thorough evaluation in the DeFi trial (NCT03785964), an international, multicenter, randomized study involving 142 patients. The trial focused on individuals whose desmoid tumors had progressed within 12 months of screening and were not suitable for surgical intervention.
Patients in the trial were randomly assigned to receive either 150 mg of nirogacestat orally twice daily or a placebo. The primary measure of efficacy was progression-free survival (PFS), assessed through RECIST v1.1 by blinded independent central review or clinical progression adjudicated by independent review.
Key Findings
The results were striking. Median PFS was not reached in the nirogacestat group, emphasizing its efficacy compared to the placebo group, which experienced a median PFS of 15.1 months. This translated into a notable hazard ratio of 0.29, signaling a significant reduction in disease progression risk for those on nirogacestat.
In terms of the objective response rate (ORR), the nirogacestat arm demonstrated a remarkable 41% response, compared to a mere 8% in the placebo group. These findings were complemented by improvements in patient-reported worst pain, favoring those receiving nirogacestat.
Administration and Adverse Effects
Nirogacestat is recommended at a dose of 150 mg orally twice daily, with or without food, until disease progression or unacceptable toxicity. Each dose comprises three 50 mg tablets. The most common adverse reactions observed include diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.
Expedited Approval Process
This significant milestone was made possible through the Real-Time Oncology Review (RTOR) pilot program, streamlining data submission, and the Assessment Aid. The application received priority review, breakthrough designation, fast track designation, and orphan drug designation, expediting the approval process for this much-needed treatment.
Reporting and Support
Healthcare providers are encouraged to report any suspected serious adverse events related to nirogacestat use through the FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient Investigational New Drug applications (INDs) for investigational oncology products, healthcare professionals can contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.
The approval of nirogacestat marks a breakthrough in the treatment landscape for desmoid tumors. Patients, their families, and healthcare providers now have a sanctioned therapeutic option that has shown significant efficacy in clinical trials. This milestone underscores the FDA’s commitment to expediting promising treatments for serious conditions, bringing hope to those affected by rare and challenging diseases. Stay updated on the latest developments by following the Oncology Center of Excellence on social media channels.
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About the author
Dr. Sajeve Thomas is a distinguished medical professional and a compassionate guide in the field of oncology. With over a decade of dedicated experience as a board-certified medical oncologist/internal medicine specialist, Dr. Thomas has become a trusted expert in the treatment of melanoma, sarcoma, and gastrointestinal conditions. Currently practicing at the renowned Orlando Health Cancer Institute, he brings a wealth of expertise to the complex and challenging world of oncology.
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