Ionis Pharma's Drug Succeeds in Late-stage Study

Ionis Pharmaceuticals said the drug to treat this rare genetic disease met the primary goal of a late-stage study. The drug donidalorsene reduced the number of seizures in patients with hereditary angioedema better than placebo. They were treated with an 80-milligram dose by subcutaneous injection every four weeks or eight weeks. 

Hereditary angioedema is a rare and life-threatening genetic disorder that causes unpredictable and recurrent severe swelling of the skin, gastrointestinal tract, upper respiratory tract, face and throat. Based on the results of the study, Ionis is preparing to submit a marketing application to the US Food and Drug Administration, the drug maker announced.

Ionis Pharmaceuticals, Inc. is a Carlsbad, California-based biotechnology company specializing in RNA-targeted drug discovery and development. The company has three commercially approved drugs: Spinraza (Nusinersen), Tegsedi (Inotersen) and Waylivra (Volanesorsen) and four drugs in clinical trials: tominersen for Huntington's disease, tofersen for SOD1-ALS, AKCEA-APO(a)-LRx for heart . and vascular disease and AKCEA-TTR-LRx for all forms of TTR amyloidosis..

The company's first and first marketed drug was fomivirsene (Vitravene, used to treat cytomegalovirus retinitis (CMV) in immunocompromised patients. It was discovered by the NIH and licensed and developed by Isis, who later licensed it to Novartis . FDA approved it for CMV for: As the first antisense drug in August 1998.