Progenics' orphan cancer drug picks up the FDA's 'breakthrough' tag

Progenics CEO Mark Baker

Progenics Pharmaceuticals' ($PGNX) in-development drug for an ultrarare form of cancer secured the FDA's breakthrough-therapy designation, promising a smoother path through the regulatory process.

The treatment, Azedra, is a radiotherapeutic that pairs a small-molecule treatment with an imaging isotope, used in small doses to spotlight tumors and then dialed up to attack them. Progenics is developing the treatment for pheochromocytoma and paraganglioma, a pair of rare neuroendocrine tumor types with no approved therapies.

The biotech is working through a Phase IIb trial in that indication and the FDA's breakthrough tag guarantees access to top agency officials as Azedra progresses. If the study is a success, Progenics is eligible for an accelerated regulatory filing and a truncated FDA review.

"Azedra has the potential to be the first approved therapy for patients with these rare and devastating tumors, and this designation will allow for a close collaboration between Progenics and the FDA" as the drug moves forward, Progenics CEO Mark Baker said in a statement.

Progenics, which markets the constipation drug Relistor with Valeant Pharmaceuticals ($VRX), is moving on from after some alarming safety data disclosed last year seemed to dim the prospects of PSMA ADC, a Phase II treatment for prostate cancer. In a trial on 70 patients, the treatment charted a modest effect on tumors but led to a spike in toxicity that some analysts said outweighed any potential benefits, and Progenics hasn't planned any late-stage development.

The biotech's most advanced asset is 1404, an imaging agent for prostate cancer slated to enter Phase III this year.

- read the statement

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