AstraZeneca Announces Orphan Drug Designation for Eplontersen in the U.S.

Healthcare stock AstraZeneca (AZN), revealed that eplontersen, formerly known as IONIS-TTR-L, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). 

Eplontersen is an investigational antisense medicine designed to treat transthyretin amyloidosis (ATTR), a systemic, progressive, and fatal disease. The medicine helps to reduce the production of transthyretin (TTR protein) for the treatment of both hereditary and non-hereditary forms of ATTR. 

Eplontersen is currently undergoing a Phase III clinical trial for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). Notably, ATTR-CM results in progressive heart failure and death within four years of diagnosis, while ATTR-PN leads to peripheral nerve damage with motor disability within five years of diagnosis. 

The medicine will be jointly developed and commercialized by biotech company Ionis Pharmaceuticals (IONS) and AstraZeneca in the U.S., while AstraZeneca will develop and commercialize it in the rest of the world, excluding Latin America. 

ODD status is granted by the FDA to medicines that are used to treat and prevent rare diseases that affect less than 200,000 people in the U.S. 

AstraZeneca is a British-Swedish multinational pharmaceutical and biotechnology company. 

Official Comments 

EVP of Biopharmaceuticals R&D at AstraZeneca, Mene Pangalos, said, “Eplontersen has the potential to be a best-in-class treatment to halt the progression of transthyretin-mediated amyloidosis and treat this fatal condition. The FDA designation further underscores the potential for eplontersen to offer new hope to this patient population currently faced with limited treatment options.” 

Wall Street’s Take 

Yesterday, Barclays analyst Emily Field maintained a Buy rating and a price target of 11,500p (38.22% upside potential) on AZN stock. 

Consensus among analysts is a Strong Buy based on 10 Buys, 1 Hold, and 1 Sell. The average AstraZeneca stock forecast of 9,750p implies 17.16% upside potential from current levels. Shares have gained 7.5% over the past year.

Smart Score  

AstraZeneca scores a 9 out of 10 from TipRanks’ Smart Score rating system, indicating that the stock has strong potential to outperform market expectations. 

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