Ionis Pharmaceuticals Receives Orphan Drug Designation from US FDA for ION582 to Treat Rare Neurological Disease

Ionis Pharmaceuticals announced that the US FDA has granted orphan drug designation to ION582 for the treatment of a rare neurological disease. The designation provides incentives for the development of medicines for rare diseases. ION582 is being developed to treat a specific form of spinal muscular atrophy. Ionis Pharmaceuticals has five marketed medicines and a pipeline in neurology, cardiology, and other areas of high patient need.

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ION582 for the treatment of a specific form of spinal muscular atrophy (SMA). This designation provides significant incentives for the development of medicines for rare diseases, including tax credits and market exclusivity [1].

ION582 is an investigational RNA-targeted antisense medicine designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS) and increase production of UBE3A protein. The FDA and European Medicines Agency (EMA) have already granted Orphan Drug designation to ION582, and the FDA has also granted Fast Track and Rare Pediatric designations to the drug [1].

The orphan drug designation for ION582 comes as the company continues to expand its pipeline in neurology, cardiometabolic disease, and other areas of high patient need. Ionis has five marketed medicines, including SPINRAZA® (nusinersen) for spinal muscular atrophy, WAINUA® (eplontersen) for hereditary transthyretin-mediated amyloid polyneuropathy, and QALSODY® (tofersen) for SOD1-ALS [1].

Ionis' progress with ION582 underscores the company's commitment to developing innovative treatments for rare and serious diseases. The company's forward-looking statements indicate that it remains focused on advancing its pipeline and delivering life-changing advances for patients [1].

References:
[1] https://www.marketscreener.com/news/ionis-receives-u-s-fda-breakthrough-therapy-designation-for-ion582-in-angelman-syndrome-ce7d59dfdf8ffe22