The FDA has granted breakthrough therapy status to Celator Pharmaceuticals’ ($CPXX) nano-powered blood cancer therapy based on the strength of results from a Phase III trial.
The regulatory agency’s designation for Vyxeos is for the treatment of adults with therapy-related acute myeloid leukemia or AML with myelodysplasia-related changes, and includes the patients enrolled in the clinical trial.
The Ewing, NJ-based company’s drug, which is also known as CPX-351, is a 5:1 formulation of the standard cancer treatments cytarabine and daunorubicin, packaged in a nona-scale delivery platform that Celator says can boost efficacy while limiting the risks of administering each drug on its own.
“We are very happy the FDA granted Breakthrough Therapy designation for Vyxeos,” Scott Jackson, Celator CEO, said in a statement. “The breadth of the designation, which includes all adults with t-AML and AML-MRC, is encouraging as AML patients are in need of advancements in treatment.”
In data the company released in March, 309 patients with AML were treated with Vyxeos versus standard cytarabine and daunorubicin therapy and the combo treatment scored a statistically significant effect on overall survival, meeting the study’s primary endpoint.
Patients in the treatment arm posted a median survival rate of 9.56 months, besting the 5.95 months observed in the other group, the company said. Vyxeos patients also demonstrated a 31% reduction in risk of death compared to the old drugs. The therapy also came through with a statistically significant improvement in response rate.
The designation is given to new medicines when the therapy has shown substantial improvement over currently available treatments on at least one clinically significant endpoint or when there is a significantly unmet medical need.
The company said it plans to submit a New Drug Application to the regulatory agency by the end of the third quarter of this year.
- here’s the release