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Ionis' (IONS) Olezarsen Gets FDA's Orphan Tag for Rare Disease

Ionis IONS recently announced that the FDA has granted orphan drug designation to its pipeline candidate, olezarsen, for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic disease.

The FDA’s orphan drug designation is bestowed on medicines in development for the treatment, prevention or diagnosis of a rare disease that affects fewer than 200,000 people in the United States at the time of designation. The rare disease, FCS, is characterized by extremely high triglyceride levels and recurrent acute pancreatitis (AP) attacks.

Olezarsen already enjoys FDA’s Fast Track designation for the treatment of FCS.

In September 2023, Ionis released positive data from the phase III BALANCE study on olezarsen in the FCS indication.  The data showed that treatment with olezarsen led to significant triglyceride-lowering and substantial reductions in AP attacks in patients with FCS.


Management intends to file a new drug application (NDA) for olezarsen in FCS with the FDA this year based on results from the BALANCE study. If approved, olezarsen will be Ionis’ first medicine that it will launch independently. Ionis is also developing olezarsen for severe hypertriglyceridemia (SHTG) in phase III studies.

Ionis’ stock has risen 18.8% in the past year against a decrease of 5.2% for the industry.


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Olezarsen is part of Ionis’s wholly owned pipeline. Some of its other wholly owned pipeline candidates are ulefnersen and donidalorsen, which are also in late-stage development. While ulefnersen is being developed for the treatment of amyotrophic lateral sclerosis (ALS) in patients with mutations in the Fused in Sarcoma (FUS) gene, donidalorsen has been developed for treating hereditary angioedema (HAE). Ionis plans to file an NDA for donidalorsen soon.

Ionis also has a broad pipeline of partnered programs with Biogen BIIB, AstraZeneca AZN, Novartis NVS and GSK, among other large drugmakers.

Ionis has licensed Spinraza to Biogen, which is responsible for commercializing the drug approved for treating spinal muscular atrophy (SMA) worldwide. Ionis receives royalties from Biogen on Spinraza’s sales. Biogen has also expanded its collaboration with Ionis to identify new gene therapies for the treatment of SMA as well as a broad range of neurological diseases. Qalsody is the second marketed drug born under this partnership.

Ionis and partner AstraZeneca’s Wainua (eplontersen) was approved by the FDA in December for treating patients with hereditary transthyretin-mediated amyloid polyneuropathy, commonly called hATTR-PN or ATTRv-PN.

AstraZeneca and Ionis will market Wainua for ATTRv-PN in the United States, while AstraZeneca has exclusive rights to commercialize Wainua outside U.S. markets. Applications seeking approval of eplontersen for ATTRv-PN are under review in the EU and some other countries.

AstraZeneca and Ionis are also developing eplontersen for another form of amyloidosis called cardiomyopathy caused by hereditary TTR amyloidosis (ATTR-CM).

Pelacarsen is being developed in partnership with Novartis for cardiovascular disease due to elevated Lp(a) levels. Along with GSK, the company is evaluating bepirovirsen as a potential treatment for patients with chronic hepatitis B virus in two ongoing late-stage studies.

Zacks Rank

Ionis currently has a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Ionis Pharmaceuticals, Inc. Price and Consensus

Ionis Pharmaceuticals, Inc. Price and Consensus
Ionis Pharmaceuticals, Inc. Price and Consensus

Ionis Pharmaceuticals, Inc. price-consensus-chart | Ionis Pharmaceuticals, Inc. Quote

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