The FDA has granted orphan drug and rare pediatric disease designations to Spirovant Sciences’s SPIRO-2101, an inhaled adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis, the company said. SPIRO-2101 works by replacing defective cystic fibrosis transmembrane conductance regulator (CFTR) genes and is meant for CF patients who are unable to benefit from currently approved CFTR modulators, including patients with Class 1 mutations.
In addition to the inhaled AAV gene therapy, Spirovant is also developing an inhaled lentivirus vector gene therapy for CF, SPIRO-2102, which is in preclinical development. The company is part of Sumitovant Biopharma, which was formed when Sumitomo Dainippon Pharma acquired five companies from Roivant in December 2019. That group also includes Altavant Sciences, which is developing an inhaled interleukin-1 receptor antagonist for the treatment of bronchiolitis obliterans syndrome.
Spirovant Sciences CEO Joan Lau commented, “Cystic fibrosis is an incredibly debilitating and devastating disease. 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.”
Read the Spirovant Sciences press release.
