PTC Therapeutics, Inc. and Genzyme, a Sanofi company, announced today the restructuring of their collaboration. Under the original agreement, PTC held commercial rights for the U.S. and Canada and Genzyme held commercial rights in all other countries. Under the restructured agreement, PTC regains worldwide rights to ataluren and Genzyme retains an option to commercialize ataluren in indications other than nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) outside the U.S. and Canada. PTC will continue to conduct worldwide development in all indications with a near-term focus on late-stage programs in nmDBMD and nonsense mutation cystic fibrosis.
"We have been very pleased with our long-standing relationship with PTC," said David Meeker, M.D., Chief Operating Officer, Genzyme. "The decision to restructure this agreement is a result of a recent portfolio assessment, and will enable us to intensify our focus on other areas of development within Genzyme where we believe we have the best ability to develop therapies to benefit the rare disease community. However, our option to reengage the collaboration reflects our belief in the potential of this approach for the treatment of nonsense mutation genetic disorders. While we will no longer be involved in advancing ataluren in nmDBMD, we remain committed to the neuromuscular community." "PTC and Genzyme share a strong commitment for bringing treatments to patients with rare and life-threatening genetic disorders," said Stuart Peltz, Ph.D., President and Chief Executive Officer, PTC Therapeutics, Inc. "We have benefited greatly from our partnership with Genzyme and we look forward to rapidly advancing the development of ataluren."