CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) has received confirmation from the U.S. Food and Drug Administration (FDA) that its New Drug Application (NDA) for donidalorsen is under review. Donidalorsen is an investigational RNA-targeted medicine intended for the prophylaxis of hereditary angioedema (HAE) attacks in patients aged 12 and older.
The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of August 21, 2025. If approved, donidalorsen would be the second independent commercial launch for Ionis, following positive results from Phase 3 and Phase 2 studies. The drug aims to reduce the production of prekallikrein (PKK), a key player in the pathway leading to HAE attacks, which are characterized by severe swelling in various body parts and can be life-threatening.
The NDA for donidalorsen includes data from the pivotal Phase 3 OASIS-HAE and OASISplus studies, as well as ongoing Phase 2 open label extension (OLE) studies. These trials have reportedly shown significant and sustained reductions in HAE attack rates, with a 96% mean reduction maintained up to three years in the Phase 2 OLE study.
Donidalorsen was generally well-tolerated in the studies, with no serious treatment-emergent adverse events related to the drug. The most common adverse event was injection site reactions, which were mostly mild or moderate.
HAE affects over 20,000 patients in the U.S. and Europe, and current treatments often involve prophylactic approaches to prevent or reduce the severity of attacks. Ionis CEO Brett Monia expressed confidence in donidalorsen's potential to improve the prophylactic treatment landscape for HAE patients.
The FDA previously granted Orphan Drug Designation to donidalorsen in 2023. Additionally, Otsuka holds exclusive rights to commercialize the drug in Europe and the Asia Pacific region and is preparing to submit a Marketing Authorization Application to the European Medicines Agency (EMA) this year.
This report is based on a press release statement from Ionis Pharmaceuticals, Inc. and does not include any speculative or promotional content. The information provided is factual and presented to inform readers about the current status of donidalorsen's regulatory review process.
In other recent news, Ionis Pharmaceuticals has been making significant progress in its product development and receiving positive analyst ratings. The company has seen positive results from a study involving a higher dose of nusinersen, a treatment for spinal muscular atrophy (SMA), which has been endorsed by Laidlaw with a Buy rating and a price target of $68.00. TD Cowen also reaffirmed a Buy rating and a price target of $59.00 for Ionis, based on recent data on Spinraza, another of the company's SMA treatments.
Ionis has also reported advancements in the development of its investigational medicine, donidalorsen, for the treatment of hereditary angioedema. Additionally, the FDA has granted Fast Track designation to zilganersen, a potential treatment for the ultra-rare neurological disorder, Alexander disease.
The company has announced a proposed public offering of its common stock, expected to amount to $500 million, to support commercial launches, clinical programs, research, and development. Financially, Ionis has reported steady results in line with annual guidance and launched two new treatments, WAINUA for hereditary ATTR polyneuropathy and QALSODY for ALS.
RBC Capital maintained its Outperform rating and $70.00 price target on Ionis Pharmaceuticals, emphasizing the company's potential in various therapeutic areas. These are all recent developments in Ionis Pharmaceuticals' operations.
InvestingPro Insights
As Ionis Pharmaceuticals (NASDAQ:IONS) awaits the FDA's decision on donidalorsen, investors should consider some key financial metrics and expert insights from InvestingPro. The company's market capitalization stands at $6.03 billion, reflecting investor expectations for its pipeline, including donidalorsen.
InvestingPro data shows that Ionis has demonstrated strong revenue growth, with a 29% increase in the last twelve months as of Q2 2024. This growth trajectory aligns with the potential commercial launch of donidalorsen, which could further boost the company's top line if approved.
However, an InvestingPro Tip cautions that analysts anticipate a sales decline in the current year. This projection underscores the importance of donidalorsen's approval for Ionis's future revenue streams. Another InvestingPro Tip notes that the company is not profitable over the last twelve months, with a negative gross profit margin of -12.86%. This financial situation emphasizes the significance of successful drug launches to improve profitability.
For investors seeking a deeper understanding of Ionis's financial health and growth prospects, InvestingPro offers 5 additional tips, providing a more comprehensive analysis of the company's position in the pharmaceutical industry.
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