Lysogene, from Paris (France) has been granted Orphan Drug Designation from the US FDA for its latest candidate for the super rare Lysosomal Storage disease Sanfilippo A Syndrome.
The phase I/II candidate has also been given Rare Paediatric Disease Designation by the FDA. This means that as Sanfilippo A Syndrome (also known as MPS-III) is a metabolic disease which affects fewer than 200,000 children in the US, Lysogene has been given a ‘Priority Review Voucher’, to be redeemed at a later date. This could make a huge difference to current patients awaiting a new therapy, seeing as the disease has a median survival rate of just 13 years old…
We interviewed Karen Aiach, CEO of Lysogene, just earlier this Month – a remarkable woman who founded Lysogene in 2009 after her daughter Ornella was diagnosed with this deadly disease. With seed funding from the French investor Sofinnova, Karen quickly rallied top experts in the field (such as Senior Scientific Officer Olivier Danos) to join her in her fight, despite coming from a non-scientific background. Indeed, Ornella is also partaking in the trials.
The candidate LYS-SAF-302 is a gene therapy which is delivered via injection directly into the brain, carried via an Adeno-associated viral (AAV) vector. This means the replacement gene ‘transfects’ the tissue in order to help it express the healthy enzyme needed to stop the progressive neurological symptoms.
As Karen explained, this milestone is great news for the clinical stage biotech as they ‘advance this product into an upcoming, multi-national phase II/III clinical trial‘. The second study for Lysogene is due to start in 2017, enrolling up to 20 patients (a dramatic increase from the 4 who partook in the last phase I/II trial – which included Ornella).
This is an exciting achievement for Lysogene and children with Sanfilippo A Syndrome, and we will continue to follow Karen’s progress closely.