Raffi Tachdjian, MD, Associate Clinical Professor of Medicine & Pediatrics, Division of Allergy & Clinical Immunology, University of California Los Angeles, discusses a new formulation of Orladeyo (berotralstat) for the treatment of pediatric patients with hereditary angioedema (HAE).
HAE is a condition characterized by recurrent episodes of severe swelling of the skin and mucous membranes. These attacks generally become more frequent after puberty, and continue throughout life, often affecting the skin, gastrointestinal tract, and upper airway. While skin swelling can cause pain, dysfunction, and disfigurement, it is not considered dangerous. When the gastrointestinal tract is involved, this may cause symptoms such as nausea, vomiting, diarrhea, and abdominal pain. The upper airway is less commonly affected, but can cause upper airway obstruction, which can be dangerous. Most airway attacks resolve before complete airway obstruction. Attacks may involve one area or a combination of areas of the body and typically go away on their own within 2 to 5 days. While people with HAE have reported various triggers of attacks, common triggers for attacks include emotional stress, physical stress, and dental procedures. HAE may be caused by genetic changes in the SERPING1 gene (also called the C1NH gene) or in the F12 gene.
The U.S. Food and Drug Administration (FDA) recently approved a New Drug Application for the use of an oral pellet formulation of once-daily berotralstat for prophylactic therapy in pediatric patients ages 2 to less than 12 years with HAE. Berotralstat is a plasma kallikrein inhibitor that prevents attacks in patients with HAE. The new pellet formulation can be mixed with water or milk, or sprinkled over soft, non-acidic food.
The acceptance of this new formulation is an important development for patients under 12 years of age, who up until this point, have only had subcutaneous injection or intravenously administered targeted treatment options. As Dr. Tachdjian explains, these methods of administration can be burdensome, especially for pediatric patients. Berotralstat is now the first and only targeted oral prophylactic therapy for patients with HAE aged 2 years and above.
The original approval was supported by interim data from the APeX-P clinical trial (NCT05453968) showing berotralstat was well tolerated, demonstrated a consistent safety profile across the 2 to 12 year age gap, and resulted in early and sustained reductions in monthly attack rates. The most commonly reported treatment-emergent adverse event was nasopharyngitis.
For more information, click here.
To learn more about HAE and other rare genetic conditions, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/
