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

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.

To Read the Complete Article at Business Wire, Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Stock image by @Giovanni_Cancemi from Depositphotos