FDA Grants Cell Therapeutics Orphan Drug Status for Opaxio

Andrew Klips  |

Shares of Cell Therapeutics Inc. (CTIC) have taken a thrashing in recent years, including dropping about 60 percent in 2012 alone. Today, the company received some good news as the U.S. Food and Drug Administration granted orphan drug status for the company’s drug Opaxio (also called paclitaxel poliglumex) as an indication for glioblastoma multiforme (GBM) treatment, a malignant brain cancer.

The FDA grants an orphan drug designation to novel drugs that treat rare diseases or disorders affecting fewer than 200,000 people in the United States. Certain benefits accompany the designation including sometimes less-stringent guidelines for clinical trials an seven years of market exclusivity upon FDA approval, tax incentives and fee waivers.

According to the National Cancer Institute, glioblastoma multiforme is the most common and deadliest form of primary brain in adults. The NCI estimates that there will be 10,000 to 12,000 new cases diagnosed in the U.S. in 2012. Currently, less than 25 percent of patients afflicted with GBM survive more than two years under today’s approved therapies.

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The regulatory decision was based upon preliminary activity shown by Opaxio in combination with a standard of care (temozolamide, or TMZ, plus radiation) in a phase II clinical trial. In the study, progression-free and overall survival was encouraging among patients with GBM, including patients whose tumors expressed unmethylated MGMT, an important DNA repair enzyme, according to the Seattle, Washington-based company.

In around 55 percent of patients with GBM, MGMT is unmethylated, which decreases the efficacy of standard therapy with TMZ plus radiation therapy. Compared to the two-year survival rate of the population of patients with GBM, those with tumors having active (unmethylated) MGMT have an even shorter survival period.

A randomized trial, expected to enroll up to 120 patients, is now underway for patients with GBM with unmethylated MGMT comparing standard TMZ and radiation to OPAXIO and radiation. Seven clinical sites will be participating. Patients in the Opaxio arm will receive the drug once every week plus radiation for six weeks. Patients in the TMZ arm will receive daily oral TMZ plus radiation for six weeks. After completion of initial therapy, both arms will receive maintenance TMZ on day 1-5 and then every 28 days for up to 12 cycles for a total of 48 weeks.

The primary goals are to determine if Opaxio plus radiation enhances progression free survival and overall survival when compared with TMZ plus radiation.

Separately, Opaxio is being tested in patients with locally advanced head and neck cancer in combination with another cancer drug. Shares of CTIC closed trading on Monday at $2.23, but are climbing in pre-market trading upon the news.

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