FDA Accepts Donidalorsen NDA for Hereditary Angioedema Prophylaxis
Key Insights
The FDA has accepted Ionis Pharmaceuticals (search)' NDA for donidalorsen, a potential prophylactic treatment for hereditary angioedema (search) (HAE) in patients aged 12 and older.
Donidalorsen, an RNA-targeted medicine, aims to prevent HAE attacks, offering a novel approach to managing this rare genetic condition.
The FDA has set a Prescription Drug User Fee Act (PDUFA) date of August 21, 2025, for the decision on donidalorsen's approval.
Ionis Pharmaceuticals (search)' donidalorsen, an investigational RNA-targeted prophylaxis medicine, has received acceptance of its New Drug Application (NDA) from the FDA for the prevention of attacks of hereditary angioedema (search) (HAE) in adult and pediatric patients aged 12 years and older. The federal agency has set a Prescription Drug User Fee Act (PDUFA) date of August 21, 2025.
Hereditary angioedema (search) is a rare genetic condition characterized by recurrent episodes of severe swelling, particularly in the face, limbs, and abdomen. Current management strategies often involve on-demand treatment of acute attacks and prophylactic therapies to reduce the frequency and severity of these episodes. Donidalorsen represents a novel approach by targeting RNA to prevent HAE attacks.
Clonidine Hydrochloride Approved for ADHD Treatment
Clonidine hydrochloride (ONYDA XR; Tris Pharma) has been FDA-approved to treat ADHD (search) in patients 6 years and up. This extended-release, liquid formulation is a non-stimulant treatment option that expands the toolkit for managing this increasingly prevalent condition.
Tapinarof Cream PDUFA Date Extended
The Prescription Drug User Fee Act (PDUFA) date for Dermavant's (now part of Organon) tapinarof cream (search), 1% (VTAMA) to treat atopic dermatitis (search) in adults and children aged 2 years and up, has been extended by 3 months. The new target action date has been set for March 12, 2025.
Topline data for the topical, steroid-free, aryl hydrocarbon receptor (search) from the ADORING 3 LTE was presented at the 44th Annual Fall Clinical Dermatology Conference in October 2024.
MenACWY Vaccination Program Reduces Meningococcal Disease
Results from a decision analytical model of the entire population of the United States from 2001 to 2021 revealed the MenACWY vaccination program in the country was associated with a reduced burden of meningococcal disease (search). Results demonstrated that incidence of invasive meningococcal disease among individuals aged 11 to 23 years would have been at least 59% higher without vaccination.