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

Date: 12/06/2018

Cary, N.C., 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.1 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.2


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


About Chiesi USA

Chiesi USA, Inc., headquartered in Cary, N.C., is a specialty pharmaceutical company focused on commercialization of products for the hospital, rare disease and target office-based specialties. Key elements of the company’s strategy are to focus its commercial and development efforts in the hospital and adjacent specialty product sector within the U.S. pharmaceutical marketplace; continue to seek opportunities to acquire companies, marketed or registration-stage products and late-stage development products that fit within the Company's focus areas. Chiesi USA, Inc. is a wholly-owned subsidiary of Chiesi Farmaceutici S.p.A. For more information, visit www.chiesiusa.com.


About Chiesi Farmaceutici S.p.A.

Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused Healthcare Group, with over 80 years of experience in the pharmaceutical industry, present in 26 countries. Chiesi researches, develops and markets innovative drugs in the respiratory therapeutics, specialist medicine and rare disease areas. Its R&D organization is headquartered in Parma (Italy), and integrated with 6 other key R&D groups in France, the USA, the UK, Sweden, and Denmark to advance Chiesi's pre-clinical, clinical and registration programs. Chiesi employs more than 5,300 people. For more information, please visit www.chiesi.com



About Holostem

Holostem Terapie Avanzate, based in Modena (Italy), is the first biotechnological company entirely devoted to development, manufacture, registration and distribution of Advanced Therapies Medicinal Products (ATMPs) based on cultures of epithelial stem cells both for cell and gene therapy.


The main aim of Holostem Terapie Avanzate is to promote epithelial stem cell-based Regenerative Medicine for patients with no alternative therapeutic solutions.


Holostem Terapie Avanzate is a university spin-off founded in 2008 through the profitable union among the scientific know-how of internationally renowned researchers such as Graziella Pellegrini and Michele De Luca, the innovative spirit of the University of Modena and Reggio Emilia  and the industrial know-how of Chiesi Farmaceutici. For more information please visit http://www.holostem.com/





1 Ahmad S. Concise Review: Limbal Stem Cell Deficiency, Dysfunction, and Distress. Stem Cells Transl Med. 2012; 1(2):110-115

2 Chiesi. Request for Orphan Drug Designation GPLSCD001 in the Treatment of Moderate to Severe Limbal Stem Cell Deficiency

3 European Medicines Agency. First stem-cell therapy recommended for approval in EU; 19th December 2014. http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2014/12/news_detail_002239.jsp&mid=WC0b01ac058004d5c1

4 FDA Orphan Drug Product designation database [Internet] https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=632218 (accessed May 2018)


Holoclar® is a registered trademark of Chiesi Farmaceutici, S.p.A.

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