Endocyte Inc. will submit its ovarian cancer drug EC145 for European market approval in the third quarter of this year after the European Commission granted it orphan drug status, the company announced Tuesday.
The submission means West Lafayette-based Endocyte could have its first commercial product as early as 2013.
It has been nearly a year since European regulators first indicated to Endocyte that they would be willing to consider EC145 for market approval as an “orphan drug,” which meant they would accept a submission based only on results of a Phase 2 clinical trial, rather than waiting for the results of a larger and longer Phase 3 human trial.
European regulators were willing to grant this “orphan drug” status to EC145 because large numbers of women have ovarian cancers that do not respond to typical chemotherapy treatments. And there has been no new drug approved to treat ovarian cancer in 10 years.
“Targeting patients with a high unmet medical need is a prerequisite to pursuing this regulatory path and, by any measure, these patients fit that definition,” said Endocyte CEO Ron Ellis in a prepared statement.
The European Commission also granted orphan status to an imaging agent Endocyte has developed, called EC20, which lights up ovarian tumors in women that have a genetic variation that makes them bond hungrily to folate.
That’s important because Endocyte’s drug, EC145, is a combination of a very powerful chemotherapy agent and folate, so that it enters cancerous cells but does not enter healthy cells. That allows EC145 to be more deadly to cancer, without serious side effects, than patients can tolerate with traditional chemotherapy agents.
Women with tumors most open to folate, known as FR++, have shown the best response to EC145. In fact, Endocyte released new data Tuesday from its Phase 2 clinical trial, which showed that women with FR++ tumors showed a 62-percent decrease in risk of progression of their cancer and a 52-percent improvement in overall survival rates, when compared with women receiving chemotherapy alone.
An announcement in December that EC145, when applied to women with all types of ovrian cancer, actually led to worse overall survival, sent Endocyte’s shares plunging.
Investors were also worried then because a shortage of Doxil, an ovarian cancer drug given in combination with EC145, had forced Endocyte to stop enrolling patients in its Phase 3 clinical trial. That halt violated one of the prerequisities for Euopean approval of EC145, which sparked fears that EC145 would be significantly delayed before reaching market.
The Doxil shortage issue was solved earlier this month when the U.S. Food and Drug Administration allowed imports of the drug from Europe.
“After discussions with EU health authorities about Phase 3 trial plans and updated clinical data, including an updated overall survival analysis, we are pleased to move forward with these applications,” Ellis said.