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ProQR's QRX-421 Gets Orphan Drug Designation from FDA and EMA

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ProQR Therapeutics N.V. (PRQR - Free Report) announced that its pipeline candidate, QRX-421, has received orphan drug designation (ODD) from the FDA and European Medicines Agency (“EMA”) for Usher syndrome. In fact, QRX-421 is the third candidate in the company’s ophthalmology pipeline and the fourth drug in the broader pipeline to receive ODD from the FDA and EMA.
Currently, QRX-421 is being evaluated for the treatment of Usher syndrome due to mutations in exon 13 of the USH2A gene.

ProQR’s shares have outperformed the industry year to date. The stock has increased 10.2% compared with the industry’s gain of 9.9% in the same time frame.

We note that the FDA, through its Office of Orphan Products Development, usually grants ODD to those drugs and biologics that are being developed for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders affecting less than 200,000 people in the United States.

Benefits and incentives include a period of marketing exclusivity of seven years in the United States and ten years in the European Union upon regulatory approval for the designated indication.

Notably, ProQR has a growing ophthalmology pipeline and also includes QR-110 that is being developed for the treatment of Leber’s congenital amaurosis 10 in clinical studies (phase I/II).

Additionally, the pipeline includes three more candidates, QRX-411, which is being developed to treat another genetic mutation resulting in Usher syndrome. Markedly, QRX-411 received ODD from the FDA and EMA in July. The other two candidates are QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.

Zacks Rank & Key Picks

ProQR currently carries a Zacks Rank #2 (Buy). Some top ranked stocks in health care sector include Alexion Pharmaceuticals, Inc. , Regeneron Pharmaceuticals, Inc. (REGN - Free Report) and Aduro BioTech, Inc. . While Alexion and Regeneron sport a Zacks Rank #1 (Strong Buy), Aduro holds a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Alexion Pharmaceuticals’ earnings per share estimates have moved up from $5.32 to $5.61 for 2017 and from $6.53 to $6.92 for 2018 over the last 60 days. The company delivered positive earnings surprises in all the trailing four quarters, with an average beat of 11.12%. The share price of the company has increased 18.3% year to date.

Regeneron’s earnings per share estimates have increased from $12.86 to $14.99 for 2017 and from $15.52 to $16.49 for 2018 over the last 60 days. The company pulled off positive earnings surprises in two of the trailing four quarters, with an average beat of 10.11%. The share price of the company has increased 34.6% year to date.

Aduro’s loss estimates per share have narrowed from $1.36 to $1.32 for 2017 and from $1.26 to $1.24 for 2018 over last 30 days. The company came up with positive earnings surprises in two of the trailing four quarters, with an average beat of 2.53%.

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