Industry Insights: Healthcare
In this week’s insights post, we explore three recent drug approvals that may be groundbreaking in disease treatment.
Semaglutide gets approval for MASH with fibrosis
The US Food and Drug Administration (FDA) has granted approval to semaglutide (Wegovy; Novo Nordisk) as the first GLP-1 receptor agonist indicated for metabolic dysfunction–associated steatohepatitis (MASH) with moderate to advanced liver fibrosis. Approval was based on part 1 of the phase 3 ESSENCE trial, which showed that 63% of patients on semaglutide achieved resolution of steatohepatitis without worsening fibrosis at 72 weeks, compared with 34% on placebo. In addition, one-third achieved both resolution and improvement of fibrosis. MASH is a progressive liver disease that is closely linked to obesity and often goes undetected until advanced stages. Semaglutide joins resmetirom as one of only two FDA-approved therapies for this condition.
FDA approves the first RNA-targeted therapy for hereditary angioedema
Donidalorsen (Dawnzera; Ionis Pharmaceuticals) has become the first FDA-approved RNA-targeted prophylactic treatment for hereditary angioedema (HAE). HAE is a rare, inherited genetic disorder that causes recurrent attacks of severe, non-itchy swelling in various parts of the body, including the limbs, face and airway. The phase 3 OASIS-HAE trial demonstrated that donidalorsen reduced monthly attack rates by 81% at 24 weeks with 4-weekly dosing. Attacks were reduced by 87% after the second dose. Patients taking the therapy every 8 weeks also experienced a 55% lower attack rate than those receiving placebo. Donidalorsen can offer flexibility and long-lasting efficacy as it is administered subcutaneously via autoinjector.
Brensocatib: the first approved therapy for bronchiectasis
The FDA has authorised brensocatib (Brinsupri; Insmed) as the first approved therapy for patients living with non–cystic fibrosis bronchiectasis. Bronchiectasis is a chronic lung disease that inflames and permanently thickens the walls of the airways. Brensocatib is an oral dipeptidyl peptidase 1 (DPP1) inhibitor targeting neutrophil-driven inflammation. Results from the phase 3 ASPEN trial, the largest study conducted in this disease, showed that brensocatib reduced pulmonary exacerbation rates compared with placebo and preserved lung function. Nearly half of patients on brensocatib remained exacerbation-free at one year, versus 40% on placebo.
