These 3 drugs are set for FDA determinations soon. Here's why Optum says payers should take notice

Another gene therapy product is set for an FDA determination in short order, and this trend should make payers stand up and pay attention, according to experts at Optum.

Etranacogene dezaparvovec is a new gene therapy for adults with moderately severe or severe hemophilia B. About 6,000 people in the United States have hemophilia B, and about 60% of those cases would qualify as moderately severe or severe, according to Optum's latest drug pipeline report (PDF).

Patients with severe hemophilia B are treated with clotting factor IX (FIX) replacement therapy, and lifetime costs for that treatment could range from $21 million to $23 million. As a gene therapy, etranacogene dezaparvovec could be a one-time treatment for this disease, according to the report.

The drug is set for a late November decision date from the Food and Drug Administration, and if approved would be the third gene therapy product approved this year by the FDA. Several more are in the pipeline, and all are projected to carry a hefty one-time price tag, said Arash Sadeghi, a clinical pharmacist for Optum Rx, in an interview with Fierce Healthcare.

"It's really the first year we're seeing this gene therapy wave," Sadeghi said.

Patient advocates are watching the development of these drugs closely, Sadeghi said, and, perhaps surprisingly, may hesitate to be among the first to sign on for these novel therapies.

"They don't necessarily want to be an early adopter," he said. "They want to see how these products perform."

Etranacogene dezaparvovec also falls under another trend that Optum analytics have been tracking for some time: the wave of orphan drugs coming through the pipeline. Of the three drugs highlighted in this latest report, etranacogene dezaparvovec would enter the narrowest market if approved, Sadeghi said.

The number of orphan drugs approved in the past few years has exceeded the number of non-orphan drugs, and the costs associated with these therapies have risen in tandem.

"Gene therapies are a great example of that, almost every gene therapy is treating a very rare condition," he said. "They're the poster child for this trend."

In addition to etranacogene dezaparvovec, the report highlights sparsentan, a drug originally slated for a fall determination that has been pushed back to early 2023. While this drug, a therapy for a kidney condition called Berger's disease, it also falls under orphan status.

If it secures approval in February, the drug could potentially be the first non-steroid approved for this condition, which could make it attractive to patients, according to the report.

The final drug highlighted in the report is pegcetacoplan, which is set for an FDA decision in late November. The drug would treat geographic atrophy, an eye disease that leads to loss of vision in the center of the field of view.

There are now FDA-approved drugs for this condition on the market, and that's why Sadeghi expects it would grab the highest demand of the three therapies included in this quarter's report. The drug could generate more than $900 million in revenue by 2032, if approved, Optum said.