FDA Approves Ogsiveo as the First and Only Treatment for Rare Desmoid Tumors

The FDA has approved Ogsiveo (nirogacestat) as the first treatment for desmoid tumors, offering significant benefits to patients. Developed by SpringWorks Therapeutics, Ogsiveo showed a 71% reduction in disease progression or mortality in the Phase 3 DeFi trial. It is the first FDA-approved treatment specifically for desmoid tumors.

The U.S. Food and Drug Administration (FDA) has marked a significant milestone in the treatment of desmoid tumors with the approval of Ogsiveo (nirogacestat), the first drug specifically sanctioned for this purpose. Desmoid tumors, also known as aggressive fibromatosis, are non-cancerous but can aggressively invade surrounding tissues and organs, often leading to severe pain, disfigurement, and decreased quality of life. These tumors are most commonly diagnosed in adults aged 20 to 44, with a higher prevalence in women.

“The FDA continues to address unmet medical need and advance the development of safe and effective therapies for the millions of Americans whose lives are affected by rare tumors,” said Richard Pazdur, M.D., director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Desmoid tumors are rare tumors that can lead to severe pain and disability. Today’s approval will offer the first approved treatment option for patients beyond surgery and radiation.”

The approval of Ogsiveo, is based on the results of the Phase 3 DeFi trial. This global, randomized, double-blind, placebo-controlled trial involved 142 patients, showing that Ogsiveo reduced the risk of disease progression or mortality by 71% compared to placebo. The median progression-free survival (PFS) for patients treated with Ogsiveo was significantly longer than those receiving a placebo, highlighting its efficacy in slowing the disease's progression.

“Our team is honored to deliver the first FDA-approved therapy for patients with desmoid tumors. This community has been waiting for an effective treatment that not only shrinks their tumors but also significantly improves pain, which is the most debilitating symptom reported by people living with desmoid tumors,” said Saqib Islam, Chief Executive Officer of SpringWorks.

Despite its effectiveness, Ogsiveo's journey to FDA approval was not without challenges. The drug, initially developed for breast cancer, demonstrated potential in treating desmoid tumors during early clinical trials at the National Cancer Institute. Following these promising results, SpringWorks pursued further development after separating from Pfizer. The FDA's Priority Review and Fast Track, Breakthrough Therapy, and Orphan-Drug designations facilitated Ogsiveo's approval process, emphasizing its significance in treating a rare and challenging condition.

Ogsiveo's safety profile has been generally manageable, with the most common side effects including diarrhea, ovarian toxicity, rash, and nausea. However, approximately 42% of patients experienced adverse events leading to dose reductions. The drug’s development history reflects a strategic shift in focus, from its initial design for breast cancer treatment to its current role in addressing a rare, yet debilitating, type of tumor.

Ogsiveo's approval heralds a new era for patients with desmoid tumors, offering hope where limited options existed. It underscores the importance of continuous research and development in the medical field, particularly for rare conditions that have historically been underserved by the pharmaceutical industry.