The US Food and Drug Administration (FDA) has approved the oral pellet formulation of once-daily Orladeyo (berotralstat) for prophylactic therapy in patients ages 12 years and older with hereditary angioedema (HAE).
HAE is a rare 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. Triggers for these episodic HAE attacks vary but may include emotional and physical stressors, including dental procedures. HAE may be caused by genetic changes in the SERPING1 gene (also called the C1NH gene) or in the F12 gene. In some cases, the genetic cause is not yet certain.
Berotralstat is the first and only oral therapy designed specifically to prevent attacks of HAE by decreasing the activity of plasma kallikrein. The new 150 mg pellet formulation provides an alternative administration option for patients who may have difficulty swallowing, prefer not to take a capsule, or choose to remain on a pellet formulation beyond age 12.
The pellet formulation is sprinkle-like in appearance and size and can be poured directly into the mouth and swallowed immediately with water or milk, or sprinkled over a spoonful of soft, non-acidic food.
The compound was approved in 2020 based on results across two clinical studies. Berotralstat resulted in sustained attack prevention in patients 12 years and older with a mean attack-rate reduction of 91% at 96 weeks and an 88% reduction in rescue medication use. The treatment was safe and well tolerated in both trials. The most frequently reported adverse reactions in treated patients compared with placebo were gastrointestinal reactions and back pain. These gastrointestinal reactions generally occurred early after initiation of treatment, became less frequent with time, and typically self-resolved.
For more information, visit https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-us-fda-approval-orladeyor-150-mg-oral-pellets
To learn more about HAE and other rare genetic conditions, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/