Orladeyo (berotralstat) is a prescription medication used to prevent attacks of hereditary angioedema (HAE), a rare genetic disorder characterized by sudden and recurrent episodes of swelling, inflammation, and pain in various parts of the body, including the face, lips, throat, and abdomen. It was approved by the U.S. Food and Drug Administration (FDA) in December 2020 and is manufactured by BioCryst Pharmaceuticals.
How does Orladeyo work?
Orladeyo is an oral medication that inhibits the activity of an enzyme called plasma kallikrein, which is involved in the production of bradykinin, a peptide that causes blood vessels to become dilated and permeable, leading to edema (swelling) and inflammation. By blocking plasma kallikrein, Orladeyo reduces the amount of bradykinin that is produced, thereby preventing the onset of HAE attacks.
Clinical trials for Orladeyo
The approval of Orladeyo by the FDA was based on the results of clinical trials that showed the drug to be effective at preventing HAE attacks in patients with the condition. In one study, Orladeyo reduced the rate of HAE attacks by 44% compared to placebo. Patients who took Orladeyo also had a longer time to their first HAE attack and fewer attacks that required rescue medication.
Side effects of Orladeyo
The most common side effects of Orladeyo are gastrointestinal symptoms, including nausea, abdominal pain, diarrhea, and vomiting. These side effects were generally mild to moderate in severity and resolved on their own without discontinuation of the medication. Rare but serious side effects of Orladeyo include thromboembolic events (blood clots) and allergic reactions.
Orladeyo and other HAE medications
Orladeyo is not intended to replace other HAE medications, such as plasma-derived or recombinant C1 esterase inhibitor (C1-INH) or kallikrein inhibitor medications, which have been used for many years as prophylaxis (preventative) and treatment for HAE attacks. Instead, Orladeyo is a new option for HAE patients who may prefer an oral medication that can be taken daily to prevent attacks, rather than having to undergo regular infusions or injections.
Conclusion
Orladeyo (berotralstat) is a new medication for the prevention of HAE attacks that works by inhibiting the activity of plasma kallikrein, an enzyme involved in the production of bradykinin. It was approved by the FDA in 2020 and is taken orally once daily. Clinical trials have shown it to be effective and safe, with mild side effects. It is a new option for HAE patients who may prefer an oral medication to prevent attacks, rather than undergoing regular infusions or injections. Patients should consult their healthcare provider regarding the use of Orladeyo.
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