Regeneron Pharmaceuticals said on Friday the U.S. health regulator approved its drug to treat a rare blood disease.
The drug pozelimab, branded as Veopoz, has been approved to treat adult and pediatric patients 1 year of age and older suffering from the CHAPLE disease.
Veopoz – the first treatment to be approved by the U.S. Food and Drug Administration (FDA) for the life-threatening disease – will be sold in the United States at a list price of $34,615.38 per single-use vial, the company told Reuters in an e-mailed response.
The drug will be available in the third quarter of this year, it said.
According to Regeneron, the disease has fewer than 10 patients identified in the U.S. and estimates less than 100 patients with the condition worldwide.
People with CHAPLE disease have mutated CD55 gene, which regulates the body’s mechanism for destroying microbes. Without proper gene regulation, the mechanism may start attacking normal cells of the body.
With Veopoz’s approval, the pre-approval inspection issues related to the marketing application of the higher dose of its blockbuster eye disease drug Eylea has been addressed, the company said.
The regulator’s decision on the 8mg dose of Eylea is expected in the next few weeks, the company added.
The FDA had in June declined to approve the higher-dose version following an inspection at third-party manufacturer Catalent.
Analysts have said an approval of the higher-dose version could provide Regeneron a defence against rivals such as Roche’s Vabysmo.
Veopoz’s approval was backed by data from a mid-to-late stage study, in which it showed normalization of serum albumin – a disease biomarker – and improvement or no worsening of clinical symptoms in all patients.
The drug blocks the activity of a particular factor of the microbes-destroying mechanism and prevents occurrence of the CHAPLE disease.