Progenics snags FDA nod for $300K ‘ultra-orphan’ cancer-fighter Azedra

FDA
The FDA approved Azedra after extending its review period by three months. (FDA)

Progenics Pharmaceuticals’ Azedra has picked up the first-ever approvals in two rare tumor types, and it’s got a hefty price tag to match.

The radiotherapeutic drug, approved on Monday afternoon to treat life-threatening neuroendocrine cancers pheochromocytoma and paraganglioma, will roll out with a sticker of roughly $147,000 for one therapeutic dose, bringing the total cost of treatment to just under $300,000, executives said on a Tuesday conference call with investors. But Progenics’ leaders aren’t worried about payer pushback.

“I think they accept that this is an ultra-orphan drug that’s entitled to ultra-orphan-type pricing,” CEO Mark Baker said. “We’ve done extensive work on the price point for the product and we feel like it best represents our ability to maximize” access for patients and returns for shareholders,” commercial SVP Bryce Tenbarge added.

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The company also said in an emailed statement that it had "created out-of-pocket support programs for commercially insured patients to include co-pay, co-insurance, and deductibles and financial support for regional and local travel."

Still, reimbursement discussions will take some time, as will identifying appropriate patients, executives noted. “Every hospital is going to have to enter into discussions” with payers at the beginning, Tenbarge said, pointing to an eight-week period between now and when patients will start receiving therapeutic doses of the drug.

To start, Progenics will target between 20 and 25 treatment centers, for use first on those that participated in Azedra’s clinical trials. “Part of what defines those 20 to 25 centers is a familiarity with these types of therapies,” Tenbarge said of nuclear medicine. The company has hired six reps as a field force to promote the drug, and its full commercial squad numbers 15.

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

Progenics, though, is already looking ahead to future indications for the product, whose label requires patients to have a tumor-locating scan. The company believes it can take the drug into areas where “MIBG scans are going to be positive for patients, which will include many other tumor types,” Baker said.

“We’ll be in front of” the FDA “with our ideas on that,” he added.

Progenics snagged its FDA nod after a three-month delay at the regulator, announced in March. The agency extended its review of the Azedra application after Progenics submitted additional chemistry, manufacturing and controls info.