US FDA approves SpringWorks Therapeutics' non-cancerous tumor treatment

FILE PHOTO: Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo

By Pratik Jain and Christy Santhosh

(Reuters) -The U.S. health regulator on Monday approved SpringWorks Therapeutics’ drug for treating adult patients with desmoid tumors, making it the first approved treatment for this type of non-cancerous soft-tissue growth.

Shares of the company were up about 18% after the bell.

The company expects the monotherapy nirogacestat, branded as Ogsiveo, to be available in the United States within 10 days of approval, CEO Saqib Islam had told Reuters ahead of the Food and Drug Administration’s decision.

The FDA approval was supported by data from a late-stage trial, which tested 142 adults with progressing desmoid tumors, where the treatment showed a 71% lower risk of disease progression or death compared to placebo.

After two years, there was no evidence of tumors getting worse in 76% of people who received Ogsiveo, compared with 44% of people who received a placebo.

People treated with Ogsiveo also reported reduced pain and improved physical functioning, the data showed.

The drug works by blocking the activity of an enzyme called gamma secretase that helps activate a signaling protein called Notch, that is believed to drive the growth of desmoid tumors.

TD Cowen analyst Yaron Werber estimates a slow and gradual launch for the therapy, that would build especially as patients can stand therapy for a long period of time.

The brokerage estimates U.S. sales of $3 million in 2023 and $79 million in 2024 and peak sales of $544 million in 2032.

“(We are) positive that payers will recognize the value that Ogsiveo brings to patients and provide coverage,” CEO Islam said.

Desmoid tumors are rare, abnormal non-cancerous growths that occur in connective tissues and are associated with a high rate of recurrence.

An estimated 1,650 people in the U.S. are diagnosed with desmoid tumors each year, according to data from the National Institutes of Health.

(Reporting by Pratik Jain and Christy Santhosh in Bengaluru; Editing by Shilpi Majumdar and Shailesh Kuber)