LYSOGENE received US orphan drug designation for SAF-301 for treatment of MPS III A

LYSOGENE announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its lead gene therapy product SAF-301 for the treatment of MPS III A.

SAF-301 is an adeno-associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs currently under investigation in a Phase I/II clinical trial (P1-SAF-301) conducted in France.

In September 2010, the European Commission granted SAF-301 orphan designation for the same indication in the European Union.

Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to drugs intended to treat rare disease or condition affecting fewer than 200,000 people in the U.S. This designation confers special incentives to the drug developer, including tax credits on the clinical development costs, prescription drug user fee waivers and may entitle a period of seven year market exclusivity in the US upon FDA approval.

Read the full press release.

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