Avrobio’s AVR-RD-04 Gains Rare Pediatric Disease Designation Cystinosis

The FDA has granted Avrobio’s investigational gene therapy AVR-RD-04 a Rare Pediatric Disease designation for the treatment of patients with cystinosis — a genetic disorder that leads to elevated levels of cystine in the body.

AVR-RD-04 is designed to genetically modify patients’ own hematopoietic stem cells to express the gene encoding cystinosin, the protein that is critically deficient in people living with cystinosis, the company said.

The investigational therapy has generated preliminary data in a phase 1/2 clinical trial. The data suggest that the approach is well-tolerated with no drug-related adverse events reported so far.

“Clinical data to date indicate this investigational approach provides benefits in multiple tissues evaluated, including the eyes, skin, gastrointestinal mucosa and the neurocognitive system,” the company said.

AVR-RD-04 previously received orphan drug designations from both the FDA and the EMA.