CSL says drug for rare swelling disorder succeeds in late-stage study

Dive Brief:

• CSL is moving toward global regulatory filings for its experimental medicine garadacimab after a successful Phase 3 trial in patients with a rare disease called hereditary angioedema.
• The Australian biotechnology company said it plans to begin seeking approval for the drug in various markets by the end of the next fiscal year, which runs through June 30. The once-monthly injection is a first-of-its-kind medication designed to prevent the devastating swelling attacks that are associated with the disease. 
• CSL didn’t release any details from the six-month study, which compared garadacimab with a placebo, only saying that it met both its primary and secondary trial goals. The drug also “demonstrated favorable safety and tolerability,” CSL said Wednesday.

Dive Insight:

Hereditary angioedema is a disease CSL already knows well. The company currently sells one drug called Berinert to treat attacks and another named Haegarda to prevent them. Haegarda works in a different way than garadacimab and must be administered every three to four days to keep swelling episodes at bay.

The genetic condition affects about one out of 50,000 people around the world. The unpredictable attacks that come with it last for days and cause severe swelling, which can be life threatening when they affect an area of the body such as the upper airway.

Garadacimab is a monoclonal antibody that works by blocking a protein, Factor XIIa, that can initiate a series of events that lead to swelling. Garadacimab is designed to stop the cascade at its origin; other drugs aim to prevent full-scale attacks by working on targets activated during the inflammatory process.

In a Phase 2 study, garadacimab reduced the rate of attacks by between 89% and 99%, depending on the dosage administered. It has the potential to be a “transformative” medicine for hereditary angioedema, CSL’s chief medical officer, Bill Mezzanotte, said in the company’s latest press release.

A number of other companies are developing new drugs for the genetic condition as well, however, and each of them offer different potential advantages. Intellia Therapeutics, for instance, is working on a gene editing therapy that’s meant to have lifelong effects. KalVista Pharmaceuticals has two medicines for hereditary angioedema in mid-to late-stage testing, including an oral treatment. It also has a third in preclinical testing that’s similar to garadacimab. Meanwhile, Ionis Pharmaceuticals is developing an RNA treatment for the disease and began Phase 3 testing last year.

CSL said it plans to release full results from its Phase 3 study in a medical journal and at an upcoming scientific meeting. The company is also looking at the potential for garadacimab to fight other diseases, including idiopathic pulmonary fibrosis.

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