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Ionis' Alexander Disease Candidate Gets Orphan Drug Status

Zacks Equity Research
·2 min read

Ionis Pharmaceuticals, Inc. IONS announced that the FDA has granted an orphan drug designation to its investigational pipeline candidate ION373, which is being developed for the treatment of people with Alexander disease, a rare and severe neurodegenerative disease.

The FDA’s orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This designation also makes the company entitled to certain other benefits including tax credits related to clinical trial expenses and an exemption from administrative fees. Upon potential approval, the status will also make ION373 eligible for seven years of marketing exclusivity in the United States.

Notably, in February 2020, the European Medicines Agency also granted an orphan drug tag to ION373 for treating Alexander disease, which is said to affect around 500 people in the EU.

Per the press release, the debilitating Alexander disease is estimated to occur in about one of one million births. Developed by Ionis, ION373 is an investigational antisense medicine, designed to stop the mutated gene from producing excess glial fibrillary acidic protein (GFAP), which causes the disease.

Meanwhile, Ionis is developing several investigational medicines to address various neurological diseases. Apart from ION373, the company is developing ION716 for treating Prion disease, ION283 for treating Lafora disease and ION363 for amyotrophic lateral sclerosis (ALS).

We note that Ionis also has many other pipeline candidates in phase II development, which it is developing in partnership with Biogen BIIB, Akcea AKCA, Roche RHHBY or on its own. The company now focuses on its wholly-owned pipeline to boost growth. It plans to conduct six pivotal studies by this year-end. It also intends to file 10 or more new drug applications through the end of 2025, possibly leading to multiple new medicines.

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