Dive Brief:
Gene therapy developer Avrobio no longer plans to seek an accelerated approval for its experimental Fabry disease treatment in the U.S. after a recent Food and Drug Administration decision to grant full clearance to the standard drug used for decades to treat the rare metabolic disorder.
In March, the FDA converted an accelerated approval it gave to Sanofi s Fabrazyme in 2003, adding to the drug s labeling additional study results on long-term treatment as well as data from real-world use. As a result, Avrobio can t request a conditional OK for its therapy from the FDA, forcing a change of plans that will push back approval discussions until after the biotech company runs a larger clinical trial.
Published: Mar 01, 2021
CAMBRIDGE, Mass. (BUSINESS WIRE) 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.
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The toxic globules of sugar and fat slowly pile up in the body s cells, accumulating in blood vessels and in major organs like the kidney and heart.
Typically, special-purpose proteins within the cell would tear apart and break down the toxins. But for people with Fabry, a rare inherited disease, genetic mutations result in garbled, sometimes missing, instructions for constructing the right proteins.
Without the right disposal tools, cells are helpless to the inexorable buildup of the dangerous molecules. The first warning signs are as varied as they are acute: burning sensations of pain in the hands and feet, abnormally low sweat production and dark skin rashes. Over time, for those most severely affected, symptoms can become dire, leading to organ dysfunction and early death.
Shares of Avrobio were up nearly 20% in premarket trading after the company posted positive clinical data from its gene therapy trials in three different rare lysomal diseases, Fabry, Gaucher type 1 and cystinosis.