Research & Development
Lin BioScience receives Rare Pediatric Disease designation from US FDA for LBS-008
18 September 2018 -

Lin BioScience, a drug development company targeting untreatable conditions in oncology, ophthalmology and metabolic diseases, announced yesterday that the United States Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to LBS-008, a first-in-class oral therapy for the treatment of Stargardt Disease, an untreatable inherited condition that causes permanent vision loss in children during childhood and adolescence.

The Priority Review Voucher Program is intended to encourage development of therapies to prevent and treat rare paediatric diseases. If the product is approved by the US FDA for Stargardt Disease, the Rare Pediatric Disease designation qualifies the company for the Priority Review Voucher. The voucher, which can be sold or transferred to another entity, can be used by the sponsor to receive Priority Review for a future NDA or BLA submission and reduce the candidate's FDA review time to six months.

The product is a first-in-class oral therapy that prevents the build-up of toxins in the eye that cause Stargardt Disease and atrophic Age-related Macular Degeneration. The NIH's Blueprint Neurotherapeutics Network, which funded the therapy's discovery and development, offers support and funding through to the completion of Phase I clinical trials. LBS-008 received both US and EU orphan drug designation in 2017 and 2018, respectively.



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