The U.S. Food and Drug Administration (FDA) has approved an oral pellet formulation of Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2 to less than 12 years of age.
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. The most common areas of the skin that are affected include the face, hands, arms, legs, genitals, and buttocks. 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. 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 or in the F12 gene.
Berotralstat is the first and only targeted oral prophylactic therapy approved for the treatment of patients with HAE. It was originally approved in 2020 for the treatment of patients ages 12 years and older. Berotralstat is designed to prevent attacks of HAE by decreasing activity of plasma kallikrein.
The approval is supported by interim data from the APeX-P clinical trial (NCT05453968), a single-arm, open-label study evaluating long-term berotralstat for pediatric patients with HAE. Results showed that treatment with berotralstat was well-tolerated, demonstrated a consistent safety profile, and resulted in early and sustained reductions in monthly attack rates with no new safety signals. The most common treatment-emergent adverse event was nasopharyngitis.
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To learn more about HAE and other rare genetic disorders, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/