The U.S. Food and Drug Administration (FDA) has granted Cytori Therapeutics Small Business status designation for fiscal year 2017, meaning that the company will pay reduced fees for regulatory filings that may arise from initiatives like the Phase 3 trial of its Habeo Cell Therapy for hand dysfunction and Raynaud’s phenomenon associated with scleroderma.
The designation, granted by the FDA’s Division of Industry and Consumer Education (DICE), qualifies Cytori for reductions in many medical device user fees, as well as a one-time waiver of the user fee for its premarket approval (PMA) application. That approval is anticipated late this year.
“Cytori Cell Therapy benefits from being regulated as a device through the PMA process,” Tiago Girão, vice president of finance and CFO of Cytori, said in a press release. “The small business designation will substantially reduce filing fees in 2017 for our planned PMA application for HABEO Cell Therapy, our lead development candidate currently under investigation for use in scleroderma, should our STAR Phase 3 data support filing of this application,” Girão said.
The ongoing STAR Phase 3 clinical trial (NCT02396238) is a placebo-controlled study evaluating the safety and effectiveness of Habeo Cell Therapy in 88 patients with hand dysfunction and Raynaud’s phenomenon (RP) associated with scleroderma. The company expects to announce the initial results from this trial by mid-2017, once the last enrolled subject has completed his or her 48-week follow-up visit.
The Habeo Cell Therapy comprises a heterogeneous population of specialized cells, including stem, lymphatic, immune, and mesenchymal and endothelial progenitor cells, that can play a role in the healing process of patients with Raynaud’s phenomenon, as well as in other diseases.
In January, Cytori announced the broadening of its ECCS-50 scleroderma treatment to include secondary Raynaud’s phenomenon, under the brand Habeo Cell Therapy. ECCS-50 for secondary RP is based on the results of the last part of the 36-month SCLERADEC I clinical trial (NCT01813279), which showed that the treatment resulted in a 90% reduction in the Raynaud’s Condition Score, a measure of RP severity. RP symptoms also are a secondary endpoint being assessed in two other Cytori cell therapy trials: SCLERADEC II (NCT02558543, ongoing) and STAR trials.