Lysogene Receives Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA

Lysogene Receives Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA 150 150 Lysogene

Designations have been granted for LYS-SAF-302 for the potential treatment of Sanfilippo Type A


Paris, France—November 24, 2015—Lysogene, a leading, gene therapy clinical-stage biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation and rare pediatric disease designation to LYS-SAF-302, a potential treatment for patients with Sanfilippo Type A, a disease also known as mucopolysaccharidosis type IIIA (MPS IIIA). LYS-SAF-302 has the potential to replace the defective gene in the cells of Sanfilippo Type A patients, which will allow for the production of the enzyme and prevent the progressive neurological damage seen in these patients.

“Receipt of these designations strengthens Lysogene’s commitment to make a meaningful impact for patients and families affected by Sanfilippo type A in the USA,” said Karen Aiach, Founder and Chief Executive Officer of Lysogene. “This is an important step as we advance this product into an upcoming, multi-national phase II/III clinical trial”.

“We seeded Lysogene right from the beginning and have continuously backed its development throughout the years. Today, the firm has achieved an important step, and we are confident that it has the potential to bring to the medical community and to patients a market breaking technology,” said Rafaele Tordjman, Managing Partner at Sofinnova Partners.

About Orphan Drug Designation

Orphan Drug Designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About Rare Pediatric Disease Designation

The FDA defines a “rare pediatric disease” as a disease that affects fewer than 200,000 individuals in the U.S. primarily aged from birth to 18 years. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug

application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher which can be redeemed to obtain priority review for any subsequent marketing application. The Priority Review Voucher may be sold or transferred an unlimited number of times.

About Lysogene

Lysogene is a clinical stage biotechnology company pioneering in the basic research and clinical development of AAV gene therapy for CNS disorders with a high unmet medical need. Since 2009, Lysogene has established a unique platform and network, with lead products in Sanfilippo A and GM1 Gangliosidosis, to become a global leader in orphan CNS diseases.

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