AVROBIO Receives Orphan-Drug Designation from the U.S. FDA for AVR-RD-01 for the Treatment of Fabry Disease

Cambridge, Massachusetts, UNITED STATES

CAMBRIDGE, Mass., Dec. 19, 2018 (GLOBE NEWSWIRE) -- AVROBIO, Inc. (NASDAQ:AVRO) (the “Company”), a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Company’s investigational gene therapy, AVR-RD-01, for the treatment of Fabry disease. The gene therapy consists of the patient’s own hematopoietic stem cells transduced with AVROBIO’s lentiviral vector, carrying a functional version of the GLA gene that encodes α‑galactosidase A (AGA) – the enzyme that is deficient in Fabry disease. Patients with Fabry disease are currently enrolling in AVRO-RD-01-201, the Company’s Phase 2 clinical trial (FAB-201), as well as in an investigator-sponsored Phase 1 study.   

“Receiving orphan-drug designation from FDA is a positive step forward in our global strategy to advance single-dose, gene therapy as a new treatment paradigm for Fabry disease. We see a promising opportunity for gene therapy to displace today’s enzyme replacement therapies that currently leave significant disease progression, a shortened lifespan and other unmet medical needs for patients with Fabry disease,” said Geoff MacKay, President and CEO of AVROBIO. “Working with medical leaders, experts and patient advocates, we are committed to moving forward with the clinical and commercial development of our gene therapies because of the potentially dramatic benefit a single-dose therapy can have in transforming the lives of patients.”

Orphan-drug designation is granted by the FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan-drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.

About AVR-RD-01

AVR-RD-01 is an ex vivo lentiviral gene therapy being investigated as a single-dose therapy with the potential to provide durable and life-long potential therapeutic benefit for patients with Fabry disease. AVR-RD-01 is designed to employ a state-of-the-art lentiviral vector platform that is an efficient and proven gene transfer system for the integration of a functional copy of the gene into the patient’s own stem cells. In patients with Fabry disease, hematopoietic stem cells are collected from the patient, and then transduced with lentiviral vector carrying a functional version of the GLA gene that encodes α‑galactosidase A (AGA) – the enzyme that is deficient in Fabry disease – to create AVR-RD-01 gene therapy. AVR-RD-01 is then infused back into the patient with the goal of restoring normal GLA gene expression such that functional AGA enzyme is sufficiently produced by the patient’s own body.

About AVROBIO, Inc.

AVROBIO, Inc., is a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose. AVROBIO’s lentiviral-based gene therapies employ hematopoietic stem cells that are collected from the patient and then modified with a lentiviral vector to insert a functional copy of the gene that is defective in the target disease. AVROBIO is focused on the development of its gene therapy, AVR‑RD‑01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including  Gaucher disease, cystinosis and Pompe disease. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com

Forward-Looking Statements

Various express or implied statements in this release concerning AVROBIO’s future expectations, plans and prospects, including without limitation, its expectations regarding the development and the continued progress of AVROBIO’s programs, including the intended incentives conferred by orphan-drug designation, and the therapeutic potential of its product candidates, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Any forward-looking statements in this press release are based on management’s current expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIO’s product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any of AVROBIO’s ongoing or planned clinical trials, the risk that AVROBIO may not realize the intended benefits of efforts to optimize its platform, and the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIO’s product candidates, and the AVROBIO may not realize the intended benefits of orphan-drug designation conferred on its product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIO’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in AVROBIO’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, as well as discussions of potential risks, uncertainties and other important factors in AVROBIO’s subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.



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