Immusoft Corporation, a Seattle-Wash.-based cell therapy company, announced today that the U.S. Food and Drug Administration (FDA) has granted it Rare Pediatric Disease Designation (RPDD) for Iduronicrin genleukocel-T, Immusoft’s Sleeping Beauty transposon-engineered autologous plasmablasts for the expression and delivery of alpha-L-iduronidase (IDUA) to treat Mucopolysaccharidosis type I (MPS I).
“This important designation from the FDA signifies the need to bring improved treatments to patients with MPS I,” said Sean Ainsworth, Immusoft’s CEO and Chairman of the Board. “This positive interaction with the FDA highlights Immusoft’s commitment to bring its novel B cell approach to pediatric patients in need of better treatment modalities.”
The FDA grants RPDD for serious or life-threatening diseases that primarily affect children 18 years old or younger and affect fewer than 200,000 people nationwide. The designation allows recipient companies, upon approval of the subject treatment, to be eligible for a priority review voucher, which may be used to obtain what is referred to as priority review for a future submission of a New Drug Application or Biologics License Application. That, in turn, can reduce FDA review time from 12 to six months. Priority review vouchers may be used by the sponsor, or sold or transferred to a third party. Recent prices on purchases of priority review vouchers have been greater than $100 million.
MPS I, which will be Immusoft’s first indication, is a rare childhood genetic disease that affects the body’s ability to produce IDUA, an essential enzyme that helps to break down long-chain sugars inside cells. When the sugar chains cannot be broken down and disposed of, they accumulate in the cells and cause progressive damage.
Earlier this year, the FDA granted Orphan Drug Designation to Immusoft for this same treatment program. It is a designation that offers incentives to companies to accelerate treatments for rare diseases. That designation, along with the RPDD, will enable Immusoft to more effectively deploy its efforts to treat MPS I and, ultimately, to further advance its modified B cell approach as a platform to address human disease.