Rare Disease Report

FDA Grants Orphan Drug Designation to LSCD Treatment, GPLSCD01

JUNE 12, 2018
Krista Rossi
The US Food and Drug Administration (FDA) has granted orphan drug designation to Chiesi USA’s investigational product, GPLSCD01, which is a treatment of ex-vivo expanded autologous human corneal epithelial cells containing stem cells for the treatment of limbal stem cell deficiency (LSCD).

“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,” stated Alan Roberts, Vice President of Scientific Affairs, Chiesi USA, in a recent statement.

GPLSCD01 (also known as Holoclar) requires a minimum of 1 to 2 mm2 of undamaged limbus. Post-biopsy, the undamaged limbus is then cultivated in vitro to make a cell sheet of epithelium with stem cells for transplantation with the potential to regenerate and repair the patient’s eye. LSCD management and symptom relief varies with damage
In the recruiting phase 4 trial of GPLSCD01, which is expected to finish in May 2020, the primary outcome measure includes transplantation success of LSCD patients as defined as the percent of patients that experience a success of first transplantation on the basis of the degree of "superficial corneal neo-vascularization" (CNV) and "epithelial defects" at approximately 12 months from the first GPLSCD01 treatment.

Secondary measures include 1 or 2 transplantation successes as defined as the percent of patients that experience clinical successes post 1 or 2 GPLSCD01 treatments on the basis of the degree of "superficial corneal neo-vascularization" and "epithelial defects" at 12 weeks. This is in accordance with the same definition as the primary efficacy endpoint assessment.

Inclusion criteria for the trial require all participating subjects be 18 years or older with the exception of 5 pediatric patients aged 2 to 17 years. Participants must also have an LSCD diagnosis.

The trial will consist of 1 experimental arm, in which subjects will be administered a biopsy from the donor eye and an implant of GPLSCD01 treatment followed by an ophthalmologic examination, a blood sample collection, digital imaging, an ECG, quality of life questionnaires, a physical examination, and an assessment of vital signs.

“Once FDA-approved, GPLSCD01 has the potential to become an important medical therapy for LSCD patients in the United States,” added Roberts.

LSCD is a rare disease that includes either 1 or both eyes and is characterized by a lack of limbal stem cells. 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 a reduction in or complete loss of vision.

GPLSCD01 has not yet been approved or reviewed by the FDA.

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