Ariad Pharmaceuticals ($ARIA) was unable to convince the FDA to pin a "Breakthrough Therapy" designation on its experimental drug for lung cancer patients with a specific genetic mutation. The coveted status could have put the compound on an expedited development and review schedule, yet the company is progressing the program without the designation.
The Cambridge, MA-based company revealed the denial on Thursday in its quarterly earnings call with analysts, where Ariad chief scientist Timothy Clackson said that regulators passed on granting the designation for the compound AP26113 in ALK-positive patients with non-small cell lung cancer based on early clinical evidence in a small number of subjects.
Clackson's disclosure is rare because companies are not required to announce the FDA's decisions on their applications for breakthrough status. Yet this highlights the importance drug developers are placing on the designation, which the U.S. agency began granting early this year after a groundswell of patient advocacy and industry input drove regulators and lawmakers to establish the program. The goal is to deliver new drugs to patients sooner than usual if early clinical evidence points to an improvement over existing treatment for a life-threatening disease.
In March Novartis ($NVS) revealed a "breakthrough" stamp for its rival ALK-targeting drug LDK378. That the FDA rejected Ariad's contender for the designation, at least in its first try, could buttress arguments that LDK378 leads the pack of next-generation drugs for lung cancer patients with ALK mutations. And Novartis and other companies with the status aim to race to approvals based on Phase II study data.
As of July 12, the FDA had granted 24 breakthrough designations and denied 18 bids for the status out of 67 applications, Reuters reported last month. Though none of the "breakthrough" therapies has been approved, making unclear how exactly the status affects the odds of the drugs making it onto the market in a speedy fashion.
"For AP26113, although the FDA did not allow the breakthrough designation request due to short follow-up and small number of patients, our takeaway from a pre-ASCO seminar is that the FDA encourages the sponsor to re-apply when more data are available," Leerink Swann analyst Howard Liang wrote in a note yesterday.
Ariad plans to provide updated Phase I/II data on its '113 program in lung cancer at a medical meeting in late September.
- here's Ariad's quarterly earnings release