October 2022 Recap: Drug Pipeline Updates
The table provided is a review of notable updates that occurred in October 2022 for investigational products in development.
Diagnosis & Disease Information
September 2022 Recap: Drug Pipeline Updates
The table provided is a review of notable updates that occurred in September 2022 for investigational products in development.
Lanadelumab Gets Priority Review for Pediatric Hereditary Angioedema
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.
MPR Weekly Dose Podcast #130
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.
Investigational CRISPR Therapy Gets Orphan Drug Status for Hereditary Angioedema
Using CRISPR/Cas9 technology, the investigational therapy is designed to edit disease-causing genes inside the human body.
Takhzyro Prefilled Syringe Now Available for Patients With Hereditary Angioedema
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.
FDA Approves Berinert Administration Kit for Hereditary Angioedema
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 On-Demand Medication Use in Hereditary Angioedema
Berotralstat may reduce the use of on-demand medications in patients with hereditary angioedema (HAE) who were previously treated with prophylactic therapies.
Oral Hereditary Angioedema Treatment Provides Effective Bradykinin-Inhibiting Concentrations
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.
New Drug Product: Orladeyo
This slideshow shows drug information for Orladeyo. Click here for the complete Orladeyo monograph.
