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Ascendis' Dwarfism Candidate Gets Orphan Drug Status in EU

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Ascendis Pharma A/S ASND announced that the European Commission (EC) has granted an Orphan Designation to its candidate TransCon C-Type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of dwarfism. TransCon CNP is a long-acting, prodrug of CNP to be given at once-weekly subcutaneous dose.

In the EU, an Orphan Drug tag is given to the products targeting a life-threatening or chronically debilitating rare disease that affects more than 5 in 10,000 individuals for which no suitable treatment is available.

We remind investors that in February 2019, the candidate received an Orphan Drug label in the United Sates for the same indication.

Ascendis is currently evaluating TransCon CNP for the treatment of achondroplasia in the global phase II ACcomplisH study.

Shares of Ascendis have increased 1.3% year to date compared with the industry’s growth of 3.9%.


We remind investors that another company, which is BioMarin Pharmaceuticals BMRN, is also developing its pipeline candidate vosoritide for treating achondroplasia, a disease for which no drug is approved yet.

In July 2020, BioMarin filed a Marketing Authorization Application (MAA) for vosoritide to the European Medicines Agency (EMA) for treating children with achondroplasia. The EU review process is expected to commence in August.

BioMarin expects to file a similar regulatory application in the United States during the third quarter of 2020.

Although BioMarin’s candidate will take some time to be launched upon a potential approval, it is most likely to bring competition for Ascendis in the long run.

Zacks Rank & Stocks to Consider

Ascendis currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. EBS and Horizon Therapeutics Public Limited Company HZNP, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

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BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report
 
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