The approval was supported extrapolation of efficacy data from the phase 3 HELP study, along with safety and pharmacodynamic data from the phase 3 SPRING study.
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Researchers found a statistically significant 73% lower HAE attack rate per 28 days with lanadelumab 300mg taken every 2 weeks vs daily berotralstat 150mg over 48 weeks.
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.
This week, news of the FDA’s vote on AMX0035, the novel ALS treatment; CPAP and BiPAP device recalls; Orphan Drug designation for a novel CRISPR therapy; Imfinzi gains new approval; and Spevigo is approved for pustular psoriasis flares.
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.
In December 2021, the US Food and Drug Administration (FDA) approved upadacitinib (Rinvoq® [AbbVie, Inc.]), a Janus kinase (JAK) inhibitor, for the treatment of adults with active psoriatic arthritis with an inadequate response or who demonstrate intolerance to at least 1 tumor necrosis factor (TNF) blocker. Upadacitinib acts by inhibiting intracellular JAK enzymes, which transmit…
APeX -2 trial findings presented at AAAAI 2022 examine the efficacy of berotralstat, in varying doses, as an oral prophylactic for reducing HAE attacks.
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.