Press Release – FDA Grants Orphan Drug Designation to Targtex’s product

The U.S. Food and Drug Administration (FDA) granted orphan-drug designation (ODD) to TTX101 for use in patients with malignant gliomas.

Press Release

Torres Vedras, September 22nd 2023 – “The U.S. Food and Drug Administration (FDA) granted orphan-drug designation (ODD) to TTX101 for use in patients with malignant gliomas.

Gliomas are a type of brain cancer that is initiated in the cells that surround and support nerve cells (glial cells) of the brain or spine. This type of brain tumors comprises about 30 percent of all brain and central nervous system tumors and 80 percent of all malignant brain tumors, including glioblastoma (GBM), oligodendroglioma, ependymoma and astrocytomas.

TargTex S.A. is an European biotech company developing a new hydrogel-based localized therapy for the treatment of brain tumors, with a lead candidate (TTX101) for GBM. GBM is the most aggressive and most common brain tumour in adults, with an average life expectancy of 16 months.

TargTex is planning a phase 1/2a clinical trial in GBM patients, in which TTX101 is applied as adjunct to surgery after tumor excision. TTX101 was designed to release the active substance over a period of weeks to cover the wound healing period between surgery and radiotherapy.

João Seixas, CEO of TargTex, refers that “The Orphan Drug Designation highlights the significant need of an efficacious therapy for this patient population as well as the potential of TTX101 to benefit such patients. Our pre-clinical data showed that TTX101 can potentially be used as monotherapy as well as a synergistic agent in combination with other drugs such as TMZ, to treat GBM and other aggressive central nervous system tumors”.

We believe that delivering our drug locally at the time of surgery will bring a major benefit to patients. The drug shows a highly promising profile, with an ideal diffusion profile in the brain, without significant local or systemic toxicity and enabling tumor eradication in preclinical models in rodents.

Orphan Drug Designation is granted by the FDA to a drug or biological product that is developed to prevent, diagnose, or treat a rare disease or condition that affects less than 200,000 people in the United States (US). The designation qualifies sponsors for incentives such as tax credits for certain clinical trials, exemption from user fees, and the potential of 7 years of US market exclusivity after approval.