Compassionate-use pilot study shows potential in hereditary angioedema treatment

A compassionate-use pilot study evaluated the impact of IONIS-PKK-LRx (a ligand-conjugated antisense oligonucleotide designed for receptor-mediated delivery to hepatocytes) on 2 patients with severe bradykinin-mediated angioedema, according to results published in New England Journal of Medicine.

Hereditary angioedema is a rare disorder that results in severe recurrent swelling.

The patients were treated for 12-16 weeks with weekly subcutaneous injections of the unconjugated parent drug, IONIS-PKKRx. After the initial period, patients were given IONIS-PKK-LRx (80 mg) every 3-4 weeks for 7 to 8 months.

Patients saw a reduction in the angioedema attack rate.

Read the full study here.

Reference
Cohn DMC, Viney NJ, Fijen LM, et al. Antisense inhibition of prekallikrein to control hereditary angioedema.