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Limbal stem cell deficiency treatment granted orphan drug designation

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The FDA has granted orphan drug designation to GPLSCD01, an investigational product for the treatment of limbal stem cell deficiency, Chiesi USA and Holostem Terapie Avanzate announced in a press release.

GPLSCD01 requires a biopsy of undamaged limbus, which is cultivated in vitro to produce a cell sheet of epithelium containing stem cells for transplantation. This could restore the corneal epithelium without needing donor tissues, the release said.

“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,” Alan Roberts, Chiesi USA’s vice president of scientific affairs, said in the release. “Once FDA approved, GPLSCD01 has the potential to become an important medical therapy for LSCD patients in the U.S.”