ORLADEYO® (berotralstat) Gains Approval in Mexico

BioCryst Pharmaceuticals, Inc. has announced that Mexico's Federal Commission for Protection against Health Risks (COFEPRIS) has approved ORLADEYO® (berotralstat), an oral medication for preventing hereditary angioedema (HAE) attacks in individuals aged 12 and above. This approval marks the fourth country in the pan-Latin America region to approve ORLADEYO, following Chile, Argentina, and Brazil. The approval is part of BioCryst's collaboration with Pint Pharma, which is responsible for the drug's marketing and commercialization throughout the region.

Charlie Gayer, BioCryst's chief commercial officer, expressed pride in the partnership with Pint Pharma to provide a new treatment option for HAE patients in Mexico and across Latin America. Pint Pharma holds the exclusive rights to register and promote ORLADEYO in the region and is tasked with obtaining and maintaining all necessary marketing authorizations.

ORLADEYO® (berotralstat) is distinguished as the first and only oral therapy specifically designed to prevent HAE attacks. The once-daily capsule works by reducing the activity of plasma kallikrein, which is instrumental in preventing the occurrence of these attacks.

In the United States, ORLADEYO® is approved for the prophylaxis of HAE attacks in adults and pediatric patients aged 12 and older. However, it is not indicated for the treatment of acute HAE attacks, as its safety and efficacy for this use have not been established. The recommended dosage is one 150 mg capsule daily, and exceeding this dose can lead to potential QT prolongation, which is a measure of delayed heart repolarization that can increase the risk of irregular heartbeats.

Common side effects observed in patients using ORLADEYO include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. For patients with moderate to severe liver impairment, a reduced dose of 110 mg taken with food is recommended. Additionally, ORLADEYO interacts with several other medications. For instance, P-glycoprotein inducers such as rifampin and St. John’s wort can reduce berotralstat's plasma concentration, potentially diminishing its effectiveness. Therefore, such inducers are not recommended for use with ORLADEYO.

Berotralstat also acts as a moderate inhibitor of the enzymes CYP2D6 and CYP3A4 at the 150 mg dosage. Medications metabolized by these enzymes, particularly those with a narrow therapeutic index, may require careful monitoring and dosage adjustments when taken alongside ORLADEYO. At higher doses, berotralstat inhibits P-glycoprotein, necessitating similar precautions for coadministered P-gp substrates like digoxin.

The drug's safety and effectiveness for children under 12 years old remain unconfirmed. Moreover, there is limited data on the use of ORLADEYO during pregnancy and breastfeeding, leaving uncertain risks for the unborn child or breastfed infant.

BioCryst Pharmaceuticals is a biotechnology firm dedicated to developing treatments for rare diseases, with a focus on complement-mediated conditions. Their expertise in structure-guided drug design has led to the creation of ORLADEYO and a robust pipeline of small-molecule and protein therapeutics aimed at hard-to-treat illnesses.