FDA Approves First Drug Therapy for Desmoid Tumors

The U.S. Food and Drug Administration recently approved Ogsiveo™ (nirogacestat) tablets for adult patients with progressing desmoid tumors who require systemic treatment. Ogsiveo is the first drug to be approved for the treatment of patients with desmoid tumors, a rare subtype of soft tissue sarcomas.

Desmoid tumors are noncancerous but can be locally aggressive. The tumors may invade surrounding structures and organs, resulting in pain, mobility issues, and decreased quality of life. Although surgical removal has historically been the treatment of choice, there is a high risk that the tumor will return or that other health challenges will occur after removal. Therefore, systemic therapies are being increasingly evaluated in clinical trials. 

“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, MD, director of the FDA’s Oncology Center of Excellence and acting 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.”

Safety and effectiveness of Ogsiveo

The effectiveness of Ogsiveo was evaluated in an international, multicenter, randomized, double-blind, placebo-controlled trial in 142 adult patients with progressing desmoid tumors not amenable to surgery. Patients were randomized to receive 150 mg of Ogsiveo or placebo orally, twice daily, until disease progression or unacceptable toxicity. The pivotal clinical trial demonstrated that Ogsiveo provided clinically meaningful and statistically significant improvement in progression-free survival compared to placebo. 

Additionally, the objective response rate was also statistically different between the two arms with a response rate of 41 percent in the Ogsiveo arm and 8 percent in the placebo arm. The progression-free survival results were also supported by an assessment of patient-reported pain favoring the Ogsiveo arm. 

The most common side effects seen in at least 15 percent of the patients in the trial were:

• Diarrhea

• Ovarian toxicity 

• Rash

• Nausea 

• Fatigue 

• Stomatitis 

• Headache

• Abdominal pain

• Cough

• Alopecia

• Upper respiratory tract infection

• Dyspnea 

Ogsiveo was granted Priority Review under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition compared to available therapies. Ogsiveo also received FDA Fast Track, Breakthrough Therapy, as well as Orphan Drug designations for treating desmoid tumors.

- This press release was originally published on the U.S. Food and Drug Administration website