FDA grants ‘Breakthrough’ status for Celator’s blood cancer drug

FDA sign

Celator Pharmaceuticals ($CPXX) has been given a “Breakthrough Therapy” designation for its experimental blood cancer drug Vyxeos.

The drug, also known as CPX-351, has recently posted positive results from a pivotal Phase III trial in older patients with previously untreated high-risk acute myeloid leukemia (AML).

The FDA’s designation--which speeds up its review of a new medicine--is for patients with therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

The Phase III trial met its primary endpoint by showing statistically significant improvement in overall survival; detailed data will be presented at ASCO next month.

Its current data for the drug showed median overall survival for patients treated with Vyxeos was 9.56 months compared to 5.95 months--a 3.61-month improvement in favor of Celator’s treatment against a chemotherapy agent known as 7+3.

The percentage of patients alive a year after randomization was 41.5% on the Vyxeos arm compared to 27.6% on the chemo arm, with the percentage of patients alive two years after randomization being 31.1% on the Vyxeos arm, compared to 12.3% on the 7+3 arm.

These data are particularly impressive considering that AML has a dismal survival rate, and fewer than 10% of patients with high-risk forms of the disease live more than 5 years after diagnosis, according to Celator.

The drug is a 5:1 formulation of the standard cancer treatments cytarabine and daunorubicin, packaged in a nano-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,” said Scott Jackson, CEO of the small NJ-based biotech, in a release. “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. We look forward to working with the FDA to bring Vyxeos to patients as quickly as possible.”

The FDA awards its Breakthrough Therapy designation in order to quicken the development and review of new medicines for life-threatening diseases. Typically, a new drug must show a substantial improvement over other available therapies on at least one clinically significant endpoint to gain this designation.

Celator said it plans to submit an NDA to the FDA by the end of the third quarter. The biotech saw its shares jump by over 10% at the end of play yesterday on the news.

- check out the release

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