Gene therapy specialist Spirovant Sciences, which is ultimately owned by Japan’s Sumitomo Dainippon Pharma (TYO: 4506), says that the US Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease designations to its lead product candidate, SPIRO-2101, for the treatment of cystic fibrosis.
SPIRO-2101, an inhaled adeno-associated virus (AAV) gene therapy, is designed to replace a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene in patients with class 1 mutations or in those who are unable to tolerate an existing CFTR modulator. SPIRO-2101 contains an evolved AAV capsid engineered to have high tropism to human airway epithelia. This treatment is designed for these patients who currently have no approved modulator therapies.
"Cystic fibrosis is an incredibly debilitating and devastating disease," said Joan Lau, chief executive of Spirovant Sciences, adding: "Receiving Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 underscores the urgency for a treatment option for these patients, particularly those with very rare and more severe types of cystic fibrosis, the nonsense mutation subtype. With these important regulatory milestones, combined with our amazing and growing team, we are well positioned to advance SPIRO-2101 into the clinic and offer hope to these patients."
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