"I wonder if the delays in PTX-100 have anything to do with this...

"I wonder if the delays in PTX-100 have anything to do with this rare disease endpoint advancement (RDEA) pilot program?"

Every possibility... and if so, it has given us the huge advantage of bolstering the data with more clinical responses in the interim! Another patient with complete eradication of cancer was reported in the recent Qrtly.

The PTX-100 Ph 1b data exceeds that of the Tecelra trial which has received an Accelerated Approva pathwayl. While it may not be appropriate to compare unrelated trials (without considering the SOC), the list of side effects associated with Tecelra is extensive. PTX-100 has an excellent safety profile with just a few low-grade side effects. So, its looking more and more likely that PTX-100 will qualify.

To Find Out More Information about FDA’s Rare Disease Programs:

Director of Strategic Coalitions for the Hub (Associate Director for Rare Disease Strategy)

Rare Diseases at FDA For Patients
CBER Rare Disease Program
CDER’s Rare Diseases Team
Accelerating Rare disease Cures (ARC) Program
FDA Voices: FDA Helps to Deliver Meaningful Advances for Rare Disease Patients
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies