ODA-designation application to approval in 19 days for Curtana Pharmaceuticals

It’s not certain that it’s a record-setting pace, but the success of Curtana Pharmaceuticals, a Texas startup, in winning orphan drug designation is one of the fastest ever. The action comes shortly after FDA Commissioner Scott Gottlieb announced a new FDA effort to speed up Orphan Drug Act (ODA) designations; he committed the agency to reduce review times from 180 days to 90 or less, and set up a go-team to clear out a backlog of some 200 applications that had been pending. (Did it help that the disease—gliomas and glioblastomas—Curtana is targeting is the very disease Senator John McCain is suffering? These days in Washington, who knows?)

ODA designation itself doesn’t necessarily get a drug through the approval process faster, but it does “highlight the potential value” of the drug, according to Laurie Halloran, CEO of Halloran Group, which is the company Curtana worked with to submit the ODA application. ODA designation can accelerate finding clinical trial patients when the testing reaches that stage; serves to reassure early-stage investors in a startup, and gets that startup experienced in developing the clinical data FDA will ultimately look for.

Curtana’s lead product, CT-179, targets glioma cancer stem cells; when combined with temozolomide and radiation (conventional therapies), the agent is believed to inhibit tumor growth. Besides the fast review, FDA also took the step of not requiring CT-179 to be tried as a monotherapy first. “This is a much-needed departure from the typical development process, which is to first test a new drug as monotherapy in patients with recurrent disease, a strategy that provides safety data, but is frequently unsuccessful in providing adequate evidence of activity," said Gregory Stein, CEO of Curtana, in a statement.

In parallel with the ODA application, Curtana has held a pre-IND meeting with FDA, which provided “general acceptance” of Curtana’s Phase I plan, according to Stein. The company expects to file its IND application in the first half of 2018.

Halloran Group has handled 20 IND applications in the past few years, according to COO Greg Dombal, and roughly half of those involved ODA designations. ODA “is becoming a specialty for us,” he says.