Regeneron wins FDA approval for rare immune disease med Veopoz

Along with resolving site inspection issues that cost Regeneron a high-dose Eylea launch in June, the company has scored an FDA nod for Veopoz as the first treatment for the ultra-rare inherited immune condition Chaple disease.

The disease, which is also known as CD55-deficient protein-losing enteropathy, has a global patient population of fewer than 100 people. In just the U.S., fewer than 10 patients have been diagnosed, Regeneron said in its Friday release.

Chaple disease is caused by mutations of the complement regulator CD55 gene, which can lead the complement system to attack the body’s own cells.

Enter Veopoz, a fully human monoclonal antibody that targets a protein involved in complement system activation called complement factor C5. It’s approved for patients 1 year of age and older and comes with a $34,615 price tag per single-use vial, a Regeneron spokesperson said in an emailed statement.

The injection was tested in a phase 2/3 trial that enrolled 10 patients ages 3 to 19 years. In the trial, all of patients saw normal concentrations of serum albumin and serum immunoglobulin by week 12 and maintained those levels through at least 72 weeks of treatment.

“As the first-ever treatment for Chaple, Veopoz is a testament to our commitment to uncovering genetic insights and applying them to the development of effective treatments for patients in need—regardless of the prevalence of their disease,” Regeneron Chief Scientific Officer George Yancopoulos, M.D., Ph.D., said in the company’s release.

The company is pursuing several other clinical studies to explore Veopoz’s “broader potential," Yancopoulos added.

For one, the drug is being tested in combination with Alnylam’s cemdisiran, an siRNAi C5 inhibitor, to treat other complement-mediated disorders, including the blood disease paroxysmal nocturnal hemoglobinuria and the neuromuscular condition myasthenia gravis.

Veopoz will be commercially available for Chaple disease patients during this year’s third quarter, according to the spokesperson.

Besides the approval, Regeneron has another reason to celebrate.

In a second approval, the FDA cleared Regeneron's 8-mg Eylea. The high-dose version of the blockbuster eye drug was previously hit with a rejection after issues at third-party Catalent's plant in Bloomington, Indiana. Both Veopoz and high-dose Eylea use the same drug filler facility.

The high-dose version of Eylea will battle it out with Roche’s rival Vabysmo, which recently crossed the blockbuster threshold itself.