US FDA approves Regeneron’s ultra-rare blood disease drug

By Pratik Jain

(Reuters) -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, would treat CHAPLE disease in adult and pediatric patients 1 year of age and older.

Veopoz — the first treatment to be approved by the U.S. Food and Drug Administration for the life-threatening disease — will be sold in the U.S. at a list price of $34,615.38 per single-use vial, the company told Reuters in an emailed response.

The drug will be available in the third quarter of this year, it said.

Regeneron (NASDAQ:) said the disease has fewer than 10 patients identified in the U.S. and estimates less than 100 patients 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.

Veopoz’s approval, however, comes with a boxed warning for serious meningococcal infections, as the treatment poses risks of developing the life-threatening bacterial infection.

With Veopoz’s approval, the company said the pre-approval inspection issues related to the marketing application of the higher dose of its blockbuster eye disease drug Eylea, or aflibercept, has been addressed.

The regulator’s decision on the 8mg dose of Eylea is expected in the next few weeks, the company added.

Imminent approval would allow the company to quickly begin converting new patients to 8mg aflibercept ahead of biosimilar entrants, potentially preserve its Eylea franchise and future revenues from the high-dose version which could reach as high as $9.8 billion, according to BMO Capital Markets analyst Evan Seigerman.

Seigerman sees biosimilars for Eylea potentially able to enter the market in May 2024.

The FDA had in June declined to approve the higher-dose version following an inspection at third-party manufacturer Catalent (NYSE:).

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