Pozelimab, sold under the brand name Veopoz, is a recombinant

monoclonal antibody A monoclonal antibody (mAb, more rarely called moAb) is an antibody produced from a cell lineage made by cloning a unique white blood cell. All subsequent antibodies derived this way trace back to a unique parent cell. Monoclonal antibodie ...

used for the treatment of CD55-deficient protein-losing enteropathy, also known as CHAPLE disease. Pozelimab is a

complement Complement may refer to: The arts * Complement (music), an interval that, when added to another, spans an octave ** Aggregate complementation, the separation of pitch-class collections into complementary sets * Complementary color, in the visu ...

inhibitor. It is produced using recombinant DNA technology in

Chinese hamster ovary cell Chinese hamster ovary (CHO) cells are a family of immortalized cell lines derived from epithelial cells of the ovary of the Chinese hamster, often used in biological and medical research and commercially in the production of recombinant therap ...

s. The most common adverse reactions include upper respiratory tract infections, fractures, hives, and alopecia. Pozelimab was approved for medical use in the United States in August 2023. It is the first FDA-approved treatment for CHAPLE disease. The US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) considers it to be a

first-in-class medication A first-in-class medication is a prototype drug that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medic ...

Medical uses

Pozelimab is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for the treatment of people with CD55-deficient protein-losing enteropathy, also known as CHAPLE disease. CHAPLE—which stands for complement hyperactivation, angiopathic thrombosis, and protein-losing enteropathy—is an inherited immune disease that causes the complement system (the part of your immune system that defends the body against injury and foreign invaders like bacteria and viruses) to become overactive. It is caused by mutations of the complement regulator CD55 gene, which can lead to the complement system attacking the body's own cells. CHAPLE disease is a rare disease, with fewer than 100 participants diagnosed worldwide. Symptoms can include abdominal pain, nausea, vomiting, diarrhea, loss of appetite, weight loss, impaired growth, and edema (swelling). Severe thrombotic vascular occlusions (blockage of blood vessels) can also occur among participants with CHAPLE disease, which can be life-threatening.

Adverse effects

The US prescribing label for pozelimab has a

boxed warning In the United States, a boxed warning (sometimes "black box warning", colloquially) is a type of warning that appears near the beginning of the package insert for certain prescription drugs, so called because the U.S. Food and Drug Administratio ...

for serious meningococcal infections. The most common adverse reactions (in 2 or more patients) are

upper respiratory tract infection An upper respiratory tract infection (URTI) is an illness caused by an acute infection, which involves the upper respiratory tract, including the nose, sinuses, pharynx, larynx or trachea. This commonly includes nasal obstruction, sore throat ...

fracture Fracture is the appearance of a crack or complete separation of an object or material into two or more pieces under the action of stress (mechanics), stress. The fracture of a solid usually occurs due to the development of certain displacemen ...

urticaria Hives, also known as urticaria, is a kind of skin rash with red or flesh-colored, raised, itchy bumps. Hives may burn or sting. The patches of rash may appear on different body parts, with variable duration from minutes to days, and typically ...

, and

alopecia Hair loss, also known as alopecia or baldness, refers to a loss of hair from part of the head or body. Typically at least the head is involved. The severity of hair loss can vary from a small area to the entire body. Inflammation or scarring ...

History

The efficacy and safety of pozelimab was evaluated in a single-arm study (NCT04209634), in which participants' outcomes were compared to pre-treatment data in participants with active CD55-deficient protein-losing enteropathy who had hypoalbuminemia. Participants' diagnoses were based on a clinical history of protein-losing enteropathy symptoms and with a confirmed genotype of biallelic CD55 loss-of-function mutation. Active CD55-deficient protein-losing enteropathy was defined as hypoalbuminemia (serum albumin concentration of ≤3.2 g/dL) with one or more of the following signs or symptoms within the last six months: diarrhea, abdominal pain, peripheral edema, or facial edema. The U.S.

(FDA) granted the application for pozelimab fast track,

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

, and rare pediatric disease designations.

Society and culture

Legal status

Pozelimab was approved for medical use in the United States in August 2023. The FDA granted the application for pozelimab fast track,

priority review Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...

, and

designations.

Names

Pozelimab is the international nonproprietary name.

References

External links

* {{Authority control Monoclonal antibodies Orphan drugs Regeneron Pharmaceuticals