The table provided is a review of notable updates that occurred in September 2022 for investigational products in development.
The sBLA is supported by data from a phase 3 trial that evaluated lanadelumab to prevent acute HAE attacks in 21 patients 2 to less than 12 years of age.
Using CRISPR/Cas9 technology, the investigational therapy is designed to edit disease-causing genes inside the human body.
The prefilled syringe is provided as a 300mg/2mL (150mg/mL) ready-to-use solution for subcutaneous injection and does not require additional reconstitution or dilution.
The FDA has approved the supplemental request for the co-packaging of a convenience administration kit for Berinert (C1 esterase inhibitor, [Human]) for acute abdominal, facial, or laryngeal attacks of hereditary angioedema.
Berotralstat may reduce the use of on-demand medications in patients with hereditary angioedema (HAE) who were previously treated with prophylactic therapies.
A single dose of PHA-022121, an oral beta-2 antagonist, was associated with effective bradykinin-inhibiting concentrations within 15 minutes of administration in patients with hereditary angioedema.