(NASDAQ:PGNX) Azedra (Ultratrace I-131-MIBG) FDA approval for pheochromocytoma (pheo) and paraganglioma (para) is supported by encouraging Phase IIb data showing its ability to reduce antihypertensive medication use, experts said. However, they were divided if the trial should have also included a progression-free survival (PFS) co-primary endpoint for a more convincing FDA win.
Whilst some noted PFS would have spelled out Azedra’s direct impact on the tumour, others noted hypertension is more relevant to morbidity in a slowly progressing disease. Nonetheless, experts were reassured by positive secondary endpoint signals showing Azedra’s potential to improve overall survival (OS) and response rates.
The FDA extended Azedra’s PDUFA date by three months to 30 July, a March media release shows. A company spokesperson said Progenics submitted additional Chemistry, Manufacturing, and Controls (CMC) data at the FDA’s request, which required additional time for review.
Experts said this PDUFA extension is unlikely to be an approval barrier, with the worst case scenario being the FDA requesting even more CMC data thus further delaying approval. Some noted Azedra’s more heightened manufacturing demands compared to its off-label counterpart justifies more CMC detail.