AVROBIO Receives Orphan Drug Designation from the European Commission for AVR‑RD‑04, an Investigational Gene Therapy for Cystinosis

Rhea-AI Summary

AVROBIO, Inc (Nasdaq: AVRO) announces that the European Commission has granted orphan drug designation for its investigational gene therapy AVR-RD-04 aimed at treating cystinosis. This gene therapy utilizes the patient's own modified stem cells to produce cystinosin, addressing the protein deficiency caused by cystinosis. Currently evaluated in a Phase 1/2 clinical trial, AVR-RD-04 has also received U.S. orphan drug status. This designation offers benefits like reduced fees and potential market exclusivity, underscoring its therapeutic promise in a challenging treatment landscape.

Positive

• Granted orphan drug designation by the European Commission for AVR-RD-04.
• Potential for 10 years of market exclusivity following regulatory approval.
• AVR-RD-04 aims to significantly improve treatment for cystinosis patients.

Negative

• None.

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-04, the company’s investigational gene therapy for the treatment of cystinosis. AVR-RD-04 consists of the patient’s own hematopoietic stem cells, genetically modified to express cystinosin, the protein that is deficient in patients with cystinosis. AVR-RD-04 is currently being evaluated in a Phase 1/2 clinical trial (NCT03897361) sponsored by AVROBIO’s academic collaborator at the University of California, San Diego.¹

"People with cystinosis must often adhere to an extremely challenging treatment regimen to manage symptoms of their disease. Despite this chronic treatment, many face a significantly shortened life expectancy and require major interventions, such as a kidney transplant,” said Geoff MacKay, president and CEO of AVROBIO. “This investigational gene therapy, delivered in a single dose, is designed to enable patients to endogenously produce the protein their cells need to prevent the toxic build-up of cystine in tissues throughout the body. We’re pleased to receive orphan drug designation in recognition of the potential of this approach to improve on the standard of care for people living with this relentless lysosomal disorder.”

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-04 has also received orphan drug designation in the U.S. from the Food and Drug Administration.

About Cystinosis

Cystinosis is a rare, progressive disease marked by the accumulation of cystine in cellular organelles known as lysosomes. This buildup can cause debilitating symptoms including kidney failure, corneal damage and thyroid dysfunction, often leading to a shortened lifespan. Currently, more than 90 percent of treated cystinosis patients require a kidney transplant in the second or third decade of life. The current standard of care for cystinosis is cysteamine, a burdensome treatment regimen that can require dozens of pills per day and may not prevent overall progression of the disease.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statement

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 “aims,” “anticipates,” “believes,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “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, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, results of preclinical studies, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, the timing of patient recruitment and enrollment activities, and product approvals, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, and the expected benefits and results of our implementation of the plato® platform in our clinical trials and gene therapy programs, including the use of a personalized and ultra-precision busulfan conditioning regimen. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any 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 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 ongoing or planned clinical trials of 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 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, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our enrollment and development timelines and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating 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 AVROBIO’s most recent Quarterly Report on Form 10-Q, 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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¹ Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).

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FAQ

What is the significance of AVRO's orphan drug designation for AVR-RD-04?

The orphan drug designation by the European Commission provides AVROBIO with regulatory benefits such as reduced fees and 10 years of market exclusivity after approval.

What is AVR-RD-04 and how does it work for cystinosis?

AVR-RD-04 is an investigational gene therapy that uses genetically modified hematopoietic stem cells to produce cystinosin, the protein missing in cystinosis patients.

What is the current status of the clinical trial for AVR-RD-04?

AVR-RD-04 is currently being evaluated in a Phase 1/2 clinical trial sponsored by the University of California, San Diego.

Has AVROBIO received orphan drug designation for AVR-RD-04 in other regions?

Yes, AVR-RD-04 has also received orphan drug designation from the U.S. Food and Drug Administration.

What is cystinosis and why is AVR-RD-04 important?

Cystinosis is a rare genetic disease that leads to serious symptoms, including kidney failure. AVR-RD-04 aims to improve treatment outcomes significantly.