QBIOTICs' drug awarded Orphan Drug Designation for patients with soft tissue sarcoma

“Soft tissue sarcomas constitute a rare group of tumours comprising more than 80 subtypes that affect both adults and children. The prognosis of advanced soft tissue sarcoma patients remains unfavourable and new treatments are urgently needed,” said Dr Victoria Gordon, executive director, strategic alliances and investor relations at QBiotics.

“The FDA Orphan Drug Designation for tigilanol tiglate signals an important milestone for QBiotics, reflecting its recognition by the FDA as a potential new treatment option for this debilitating and life-threatening disease.”

QBiotics’ phase 2 clinical trial is being conducted at Memorial Sloan Kettering Cancer Center in NYC, with Edmund Bartlett, as principal investigator. 

The trial is assessing the preliminary efficacy and safety of tigilanol tiglate in patients with a range of advanced and/or metastatic soft tissue sarcomas (STS). 

Orphan Drug Designation (ODD) is a special status for drugs which are considered promising potential treatments for patients with rare diseases, being fewer than 200,000 cases per year in the U.S. 

The designation can provide for a seven-year window of exclusive marketing rights, post-approval, as well as exemption from user fees and eligibility for tax credits for qualified clinical trials. In addition to the financial benefits, it may also potentially shorten clinical development due to closer collaboration with the FDA.

Global Data estimates that there were 124,573 new cases of STS globally in 2021, with the incidence growing at 0.54% per year.