FDA Grants Lin BioScience Rare Pediatric Disease Designation for Experimental Treatment for Stargardt Disease

Rare Daily Staff

The U. S. Food and Drug Administration granted Lin BioScience Rare Pediatric Disease designation for its LBS-008, a first-in-class oral therapy for the treatment of Stargardt Disease.

Stargardt Disease is a rare, genetic condition that causes permanent vision loss in children during childhood and adolescence. This rare disease is caused by a mutation in the ABCA4 gene, which leads to the accelerated formation and accumulation of toxic vitamin A dimers in the retina that cause progressive retinal cell death and permanent loss of vision.

LBS-008 is a first-in-class oral therapy that prevents the buildup of toxins in the eye that cause Stargardt Disease and atrophic Age-related Macular Degeneration (dry AMD). The NIH’s Blueprint Neurotherapeutics Network, which funded the therapy’s discovery and development, provides support and funding through to the completion of Phase I clinical trials.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes LBS-008 eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review Voucher for $80.6 million.

LBS-008 previously received both orphan drug designation in both the United States and Europe.

September 17, 2018