An ultra-rare hereditary immune disease, CHAPLE is driven by the overactivation of the complement system, in turn causing potentially life-threatening abdominal and cardiovascular symptoms. Credit: Darko Djurin / Pixabay.

The US Food and Drug Administration (FDA) has accepted a priority review of the biologics license application (BLA) for Regeneron Pharmaceuticals’ pozelimab to treat children and adults with ultra-rare CHAPLE disease.

Currently, there are no approved treatments for CHAPLE, a life-threatening hereditary immune disease caused by an overactivation of the complement system.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The investigational, fully human monoclonal antibody, Pozelimab has been designed to block the complement factor C5 activity.

C5 is a protein that is involved in the activation of the complement system.

The antibody is developed using the company’s VelocImmune technology.

The regulatory body is expected to decide on 20 August.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The BLA submitted to the FDA is supported by the data obtained from an open-label Phase II/III trial, designed to evaluate the efficacy and safety of pozelimab in ten patients aged one year or above.

The findings showed that 100% of patients had quick and sustained normalisation of serum albumin (a disease biomarker) and improvement or no worsening of clinical symptoms at 24 weeks.

In the trial, the clinical symptoms were assessed, including the number of bowel movements per day, abdominal pain, and investigator-assessed facial and peripheral edema.

In April 2020, pozelimab was designated as a drug for a “rare pediatric disease” to treat CHAPLE by the US FDA, with Regeneron having an opportunity to get a rare pediatric disease priority review voucher if pozelimab is approved for CHAPLE.

It was also granted Orphan Drug Designation during the same time.

It is also being assessed along with Alnylam’s cemdisiran (siRNAi C5 inhibitor) to treat other complement-mediated disorders, including myasthenia gravis (MG) and paroxysmal nocturnal hemoglobinuria (PNH).

Earlier this month, Regeneron received US FDA approval for its Eylea (aflibercept) injection to treat the retinopathy of prematurity (ROP) in preterm infants.