Emactuzumab Gets Fast Track Designation in Tenosynovial Giant Cell Tumors

For patients with tenosynovial giant cell tumors and who would not benefit from surgery, emactuzumab has received fast track designation from the U.S. FDA.

Treatment with emactuzumab has received fast track designation from the United States Food and Drug Administration (FDA) for patients with tenosynovial giant cell tumor (TGCT) who are not amenable to or who would not benefit from surgery, according to a press release from Sino Therapeutics Limited.

The regulatory agency grants fast track designation to help aid in the development and review of new treatments for serious medical conditions that do not yet have effective options. This status encourages closer communication between the FDA and the company developing the treatment, which may help speed up the delivery of these important therapies to patients.

The investigative agent — a potentially best-in-class CSF-1 receptor (CSF-1R) inhibiting monoclonal antibody — is notably being evaluated in the phase 3 TANGENT clinical trial, a global, multi-center, randomized, double-blind, placebo-controlled registrational study.

“The granting of fast track designation for emactuzumab in TGCT highlights the devastating toll that this disease has on patients, as well as the critical need that remains for new treatment options,” said Dr. Elyse Seltzer, chief medical officer of SynOx Therapeutics, in the news release.

“Based on our clinical work to date, we believe that emactuzumab has significant potential to address key patient needs by offering an effective, short-course treatment with rapid onset and a durable response that allows individuals suffering from TGCT to better manage their disease and move forward with their lives. We look forward to completing the ongoing TANGENT study and progressing emactuzumab toward potential commercialization,” Seltzer emphasized.

Delving Into the Phase 3 TANGENT Trial

TGCT, also known as pigmented villonodular synovitis, primarily affects the soft tissue lining of joints and tendons. TGCT is a type of tumor that is classified as a fibrohistiocytic tumor and are subclassified as localized and diffuse types based on growth patterns, as well as tendon sheath, intra- and extra- articular forms based on location. These tumors are aggressive and locally destructive, according to the press release, causing significant joint pain, stiffness, loss of function and reduced quality of life. Although most patients with TGCT undergo surgery, more than half experience tumor recurrence within three years. Without treatment, TGCT can worsen over time and may cause joint damage, deformity, and in severe cases, may require joint fusion (arthrodesis) or amputation.

The tumor typically occurs in large joints such as the knee, hip, or ankle and is driven by the overproduction of a protein called CSF-1. The FDA’s decision to grant fast track designation for TGCT treatment was based on results from early phase 1/2 clinical trials, which showed that emactuzumab led to rapid, robust tumor reduction and had manageable side effects. Emactuzumab has also received orphan medicinal project designation from the European Medicines Agency.

CSF-1, also known as macrophage colony-stimulating factor, is a protein that plays a key role in certain immune cells, particularly macrophages Emactuzumab is a humanized IgG1 monoclonal antibody that specifically targets and blocks CSF-1R. By doing so, the agent reduces the number of macrophages in tumor tissue, helping to slow or stop tumor progression.

According to the press release, emactuzumab has demonstrated substantial efficacy in patients with TGCT. Clinical studies have reported an objective response rate of approximately 71%, with patients experiencing rapid tumor shrinkage, improved joint function, good tolerability and a manageable safety profile.

SynOx Therapeutics is currently evaluating additional indications where emactuzumab may be effective, the release concludes.

For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.