AVROBIO Receives U.S. FDA Rare Pediatric Disease Designation as a First-in-Class Gene Therapy for Gaucher Disease

AVROBIO Receives U.S. FDA Rare Pediatric Disease Designation as a First-in-Class Gene Therapy for Gaucher Disease

CAMBRIDGE, Mass.–()–AVROBIO, Inc.. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to rid people of genetic diseases for life, today announced that the U.S. Food and Drug Administration (FDA) granted the designation from rare pediatric disease to AVR-RD-02, a first-in-class investigational gene therapy that genetically modifies patients’ own hematopoietic stem cells (HSCs) to treat Gaucher disease, a rare lysosomal disorder that can cause multi-organ pathology, clinical morbidity and early mortality.

The FDA Pediatric Rare Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease may be eligible to receive a voucher for priority review of a post-marketing application for a different product. The priority review voucher can be used by the company or sold to a third party.

AVROBIO’s Gaucher disease program includes Type 1 and Type 3 Gaucher disease, which together affect approximately 30,000 patients worldwide. The company expects to provide an interim clinical data update for its Type 1 Gaucher disease phase 1/2 clinical trial, as well as an overview of the development and regulatory strategy for its Type 3 Gaucher disease program, in the fourth quarter of 2022.

About Gaucher disease

Gaucher disease is a rare inherited lysosomal disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages enlarged with these fatty substances are called Gaucher cells and accumulate mainly in the spleen, liver, and bone marrow. This results in a variety of potential symptoms, including greatly enlarged liver and spleen, bone problems, fatigue, low hemoglobin and platelet counts, and a lifetime adjusted relative risk of developing Parkinson’s disease that may be more than 20 times greater than the general population. Even with enzyme replacement therapy (ERT), the current standard of care, people with Gaucher disease often have a shorter life expectancy and can experience debilitating symptoms that significantly reduce their quality of life.


Our vision is to bring personalized gene therapy to the world. We target the root cause of the genetic disease by introducing a working copy of the affected gene into patients’ own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. Our first-of-its-kind project portfolio includes clinical programs for cystinosis and type 1 Gaucher disease, as well as preclinical programs for type 3 Gaucher disease, Hunter syndrome, and Pompe disease. Our proprietary plato® gene therapy platform is designed to scale to support clinical development and late-stage commercialization globally. Our headquarters are located in Cambridge, Massachusetts. For additional information, visit avrobio.comand follow us on Twitter Y LinkedIn.

Forward-looking statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as “targets,” “anticipates” , “believes”, “could”, “designed for”, “estimates”, “expects”, “forecast”, “goal”, “intends”, “may”, “plans”, “possible”, “potential”, “seeks,” “will,” and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements about our business strategy and the potential therapeutic benefits of our preclinical and clinical product candidates, including AVR-RD-02 for the treatment of Gaucher disease, the benefits and potential incentives provided by the FDA rare pediatric disease designation for AVR-RD-02, the potential benefits provided by the FDA rated by the FDA priority review voucher, the design, initiation, enrollment and timing of planned clinical trials, the results of preclinical or clinical trials, product approvals and regulatory pathways, our plans and expectations regarding interactions with regulatory agencies, the anticipated benefits of our gene therapy platform, including the potential impact on our commercialization activities, the timing and likelihood of success, the expected benefits, and the results of our implementation of the platform plato® in our clinical trials and gene therapy programs, and the expected safety profile of our preclinical and investigational gene therapies. Any such statement in this press release other than a statement of historical fact may be considered a forward-looking statement. The results of early-stage or preclinical clinical trials may not be indicative of the results of larger-scale or later-stage clinical trials and do not guarantee regulatory approval. You should not place undue reliance on these statements or the scientific data presented.

All forward-looking statements in this press release are based on AVROBIO’s current expectations, estimates and projections about our industry, as well as management’s current beliefs and expectations about future events as of today only and are subject to number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that one or more of AVROBIO’s product candidates may not be successfully developed or commercialized, the risk of interruption or delay in any ongoing or planned clinical trial by AVROBIO or our collaborators. , the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato® platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that previous results, such as signs of safety, activity or durability of effect, observed in preclinical trials or clinical trials, will not be reproduced, or will not be continued in ongoing or future studies or trials involving the AVROBIO’s product candidates, the risk that we may not obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates may not materialize as expected, the risks associated with our reliance on third-party vendors and manufacturers, risks related to the accuracy of our estimates of future revenue and expenses, risks related to our capital requirements and additional financing needs, risks related to clinical trials and business interruptions resulting from the outbreak of COVID-19 or similar public health crises, including that such disruptions may materially delay our enrollment and development timelines and/or increase our development costs or that data collection efforts may be affected or impacted by such crises, and the risks related to our ability to obtain and maintain intellectual property protection for our 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 materially and adversely from those contained in the forward-looking statements, see the section entitled “Risk Factors” in the most detailed information on AVROBIO. Recent quarterly report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIO’s subsequent filings with the Securities and Exchange Commission. AVROBIO expressly disclaims any obligation to update any forward-looking statement, except to the extent required by law.

Leave a Comment