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AVROBIO's Gaucher Disease Candidate Gets Orphan Drug Status

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AVROBIO, Inc. (AVRO - Free Report) announced that the European Commission ("EC") has granted orphan drug designation to its gene therapy candidate, AVR-RD-02, for treating Gaucher disease, a rare, inherited lysosomal storage disorder.

The company is developing AVR-RD-02 as a single dose treatment to address the head-to-toe manifestations of Gaucher disease, which can lead to debilitating complications throughout the body and brain. Current standard-of-care treatments are not able to address all these symptoms and may not be able to halt progression of the disease.

A phase I/II study — GuardOne — is evaluating the candidate in patients with type I Gaucher disease. Please note that AVR-RD-02 is a personalized gene therapy, which uses a patient’s own hematopoietic stem cells that are transplanted after genetical modification.

Notably, the orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 5 in 10,000 patients in European Union. The status makes AVR-RD-02 eligible for 10 years of marketing exclusivity in Europe, following an approval for the given indication. The designation also makes the company entitled to certain other benefits including reduced regulatory fees, clinical protocol assistance and research grants.

The candidate also enjoys orphan drug status in the United States, which was granted last year. In the United States, AVR-RD-02 will enjoy seven years marketing exclusivity following a potential approval.

Shares of AVROBIO have decreased 35.4% year to date against the industry’s growth of 2.3%.


Apart from AVR-RD-02, AVROBIO has a few other gene therapy candidates in its pipeline targeting different indications. A phase II study is evaluating AVR-RD-01 as a potential treatment for treatment-naïve patients with Fabry disease. An investigator-sponsored phase I/II study is evaluating AVR-RD-04 in patients with cystinosis, a rare, progressive disease marked by the accumulation of cystine in cellular organelles leading to eye complications, muscle wasting and kidney failure. The company’s another gene therapy candidate, AVR-RD-03, is in pre-clinical stage of development as a treatment for Pompe disease.

In May, AVROBIO collaborated with Magenta Therapeutics (MGTA - Free Report) to evaluate the potential utility of Magenta’s novel targeted antibody-drug conjugate candidate, MGTA-117, as a potential conditioning regimen for lentiviral gene therapies.

Zacks Rank & Stocks to Consider

AVROBIO currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. (EBS - Free Report) and Horizon Therapeutics (HZNP - Free Report) , both sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have moved north from $4.23 to $6.58 for 2020 and from $5.55 to $7.62 for 2021 over the past 60 days. The stock has skyrocketed 92.1% year to date.

Horizon’s earnings estimates have been revised upward from $1.95 to $2.86 for 2020 and from $2.91 to $4.34 for 2021 over the past 60 days. The stock has rallied 120.1% year to date.

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