Holostem and Chiesi Announce FDA Orphan Drug Designation for GPLSCD01 for treatment of Limbal Stem Cell Deficiency (LSCD)

Cary, N.C., and Modena, Italy, June 12, 2018 – Chiesi USA (key-A-zee), a specialty pharmaceutical company based in Cary, N.C., and the Italian biotech Holostem Terapie Avanzate S.r.l., announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to an investigational product, GPLSCD01, ex-vivo expanded autologous human corneal epithelial cells containing stem cells, for the treatment of limbal stem cell deficiency (LSCD).

LSCD is a rare and seriously debilitating condition affecting one or both eyes due to physical or chemical ocular burns, inflammatory diseases, and hereditary diseases. Affected patients lack cells called limbal stem cells, which are found at the edge of the cornea (the transparent layer in front of the eye), that normally function to continuously renew and repair the cornea. Left untreated, LSCD results in chronic pain, burning, photophobia (eye discomfort in bright light), inflammation, corneal neovascularisation (new blood vessels growing across the front of the eye), stromal scarring and the reduction or complete loss of vision.

“We are very pleased to receive orphan drug designation for GPLSCD01 for patients with LSCD. This is an important regulatory milestone for Chiesi USA as we continue to focus on meeting the needs of the rare disease community,” said Alan Roberts, Vice President of Scientific Affairs, Chiesi USA. “Once FDA-approved, GPLSCD01 has the potential to become an important medical therapy for LSCD patients in the U.S.”

The management and symptom relief of LSCD depends on the extent of the damage. GPLSCD01 requires a biopsy of undamaged limbus which is then cultivated in vitro to produce a cell sheet of epithelium containing stem cells for transplantation with the potential to regenerate and repair the patient’s eye. The autologous, or obtained from the same individual, therapy may offer the advantage of restoring the corneal epithelium without the need of donor tissues and subsequent immunosuppression.

The FDA’s orphan drug designation is granted to drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemption and seven-year marketing exclusivity upon FDA approval.

About GPLSCD01

GPLSCD01 is an investigational drug candidate in the United States. The effectiveness and safety of GPLSCD01 have not been reviewed or approved by the FDA.  GPLSCD01, known as Holoclar® in the European Union, is the first advanced medicinal product containing stem cells to be licensed in the European Union for the treatment of adult patients with moderate-to-severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. A minimum of 1 – 2 mm² of undamaged limbus is required for biopsy.

 Holoclar is manufactured by Holostem and commercialized by Chiesi in several European countries.