Treatment for Limbal Stem Cell Deficiency Gets Orphan Drug Designation

Chiesi USA announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to GPLSCD01 for the treatment for limbal stem cell deficiency.

Patients with limbal stem cell deficiency, a rare condition affecting one or both eyes, can experience chronic pain, burning, photophobia, inflammation, corneal neovascularization, stromal scarring, and reduction or complete loss of vision. Limbal stem cell deficiency may be caused by physical or chemical ocular burns, hereditary conditions or inflammatory diseases. 

GPLSCD01 is an investigational treatment made up of ex vivo expanded autologous human corneal epithelial cells containing stem cells. Treatment with GPLSCD01 requires a biopsy of undamaged limbus (minimum of 1-2mm²) which gets cultivated in vitro to produce a sheet of epithelium cells containing stem cells for transplantation. This autologous therapy can potentially regrow and repair the patient’s eye(s) without the need for donor tissues and associated immunosuppression. 

GPLSCD01 is currently approved in the European Union as Holocar for the treatment of adults with moderate-to-severe limbal stem cell deficiency (defined as superficial corneal neovascularization in ≥2 corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. 

For more information call (888) 661-9260 or visit ChiesiUSA.com.