SpringWorks begins Phase 3 trial of desmoid tumor treatment in adults
Category: #health  By Mateen Dalal  Date: 2019-05-21
  • share
  • Twitter
  • Facebook
  • LinkedIn

SpringWorks begins Phase 3 trial of desmoid tumor treatment in adults

The company is looking forward to enrolling patients with desmoid tumors to confirm the clinical benefits of nirogacestat

SpringWorks Therapeutics, a clinical-stage biopharmaceutical company, has reportedly announced the beginning of Phase 3 “DeFi” (Desmoid/Fibromatosis) study for evaluating nirogacestat, a selective, oral and small molecule gamma-secretase inhibitor, to treat adult patients having progressing desmoid tumors.

Seemingly, desmoid tumors are sporadic and are often disfiguring and debilitating soft-tissue tumors which can aggressively attack surrounding healthy tissues, including blood vessels joints, nerves, internal organs, and muscles. Based on their location and size, desmoid tumors can cause substantial morbidities, including internal bleeding, severe pain, debilitating loss of range of motion, disfigurement, and in rare cases desmoid tumor scan be lethal.

Sources familiar with the matter mentioned that an estimated 1,000 to 1,500 novel cases of desmoid tumors are diagnosed every year in the United States. For the treatment of desmoid tumors, there are presently no therapies approved by the U.S. FDA.

Chief Executive Officer of SpringWorks Therapeutics, Saqib Islam stated that those patients who are living with these debilitating tumors, the DeFi trial represents a significant step forward and could result in the first approved treatment for this underserved patient community.

Apparently, the company is looking forward to enrolling patients with desmoid tumors to confirm the clinical benefits of nirogacestat.

In June 2018, the FDA approved Orphan Drug designation for nirogacestat for the treatment of patients who were suffering from desmoid tumors, and in November 2018, the FDA granted Fast Track designation for nirogacestat in the treatment of adult patients with, unresectable, progressive, refractory or recurrent desmoid tumors or deep fibromatosis.

For the uninitiated, the DeFi trial is a double-blind, randomized, placebo-controlled, global Phase 3 trial for evaluating the efficacy, tolerability, and safety of nirogacestat in adult patients with progressing desmoid tumors. The study will enrol around 100 adult patients, who will receive placebo or 150 mg of nirogacestat twice daily.

Source Credit: https://www.springworkstx.com/wp-content/uploads/2019/05/DeFi-Study-Initiation-Press-Release.pdf

  • share
  • Twitter
  • Facebook
  • LinkedIn


About Author

Mateen Dalal    

Mateen Dalal

Mateen has completed his Bachelor’s degree in electronics and telecommunication engineering, post which he lent his proficiency to the industry, working as a quality and test engineer. Drawn intricately toward the field of content creation however, Mateen soon switc...

Read More

More News By Mateen Dalal

US senator pleas investigation over TikTok’s Musical.ly acquisition
US senator pleas investigation over TikTok’s Musical.ly acquisition
By Mateen Dalal

Musical.ly is a social media service which was released in 2014 in Shanghai and is incorporated in Santa Monica, California. The service was acquired by ByteDance and has been operating as TikTok for overseas markets. However, the viral social media...

Midatech initiates Phase 1 trial of a new hormonal cancer therapy
Midatech initiates Phase 1 trial of a new hormonal cancer therapy
By Mateen Dalal

Cancer treatment is one of the biggest challenges for the 21st century. There have been many advancements in the field of cancer treatment over the past few years. Clinical trial indeed plays a pivotal role in discovering new drugs, especially for tr...

DISH Network inks Multi-Year Carriage Agreement with Fox Corp.
DISH Network inks Multi-Year Carriage Agreement with Fox Corp.
By Mateen Dalal

Sports channels are one of the few types of TV broadcasts that people prefer live. No channel is more vital to sports fans than a regional sports network that views their favorite teams. However, a dispute emerged recently between Dish Network and Fo...