What is Holoclar and what is it used for?

Holoclar is a treatment containing stem cells, used to replace damaged cells on the surface (epithelium) of the cornea, the transparent membrane that lines the iris (the colored part of the eye). It is used in adult patients with moderate to severe limbal stem cell deficiency caused by ocular burns (including chemical burns). Patients with this condition do not have a sufficient number of limbal stem cells, which normally intervene in the regeneration process of the cornea, replacing the external corneal cells that are damaged and aging. Holoclar is a type of advanced therapy medicine called a 'tissue engineered product', which is a type of medicine that contains cells taken from the patient's limbus (at the corners of the cornea) and then grown in the laboratory so that they can be used to repair the surface damaged corneal. Because the number of patients with limbal stem cell deficiency is low, the disease is considered 'rare', and Holoclar was designated an 'orphan medicine' (a medicine used in rare diseases) on 7 November 2008.

How is Holoclar used?

Holoclar should only be used in a hospital setting, by a suitably trained and qualified ophthalmic surgeon, and should only be given to patients whose limbal cells have been used in the manufacture of the medicinal product. In the first phase of treatment, a small flap of healthy limbal tissue (1-ˆ "2 mm2 in size) is removed from the patient in a hospital setting and sent on the same day to the medicine manufacturer. Subsequently, the tissue cells are grown in the laboratory and frozen until the date of surgery is confirmed. Defrosted cells are used to make Holoclar by growing them on a membrane made up of a protein called fibrin. Holoclar, which is composed of both cells and membrane, is then sent back to the hospital, where it is immediately implanted in the patient's eye as part of surgery. After limbal tissue harvesting, patients should be treated with antibiotics to prevent an "eye infection". After surgery, the patient should undergo treatment with antibiotics and an appropriate anti-inflammatory medicine. Holoclar is intended for one treatment only. The treatment can be repeated if the treating physician deems it necessary. For more information, see the summary. the characteristics of the product (also included in the EPAR).

How does Holoclar work?

The active substance in Holoclar is the patient's limbal cells, which include cells taken from the surface of the cornea and limbal stem cells grown in the laboratory. Before using Holoclar, the damaged tissue must be removed from the corneal surface of the affected eye. Once implanted in the eye, the Holoclar corneal cells help replace the surface of the cornea, while the limbal stem cells act as reservoirs for new cells. continuously regenerate the cornea

What benefit has Holoclar shown during the studies?

As part of a retrospective study with past clinical data, Holoclar has been shown to be effective in regenerating a stable corneal surface in individuals with moderate to severe limbal stem cell deficiency caused by burns. One year after implantation with Holoclar , the implant was considered successful in 75 of 104 patients studied (equal to 72%), based on the presence of a stable corneal surface without surface defects and without or with limited invasion of new blood vessels (a typical feature limbal stem cell deficiency). There were also reductions in patients' symptoms, including pain and inflammation, as well as improvements in vision.

What is the risk associated with Holoclar?

The most common side effect with Holoclar (which may affect more than 1 in 10 people) is blepharitis (inflammation of the eyelid). For the full list of side effects and limitations, see the package leaflet.

Why has Holoclar been approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) considered that Holoclar treatment was effective in restoring healthy corneal surfaces in patients with moderate to severe limbal stem cell deficiency caused by burns as well as improving symptoms and vision. The Committee is of the opinion that moderate to severe forms of stem cell deficiency represent serious conditions which, if left untreated, can lead to severe reduction, or total loss, of vision. Side effects of treatment with Holoclar are manageable, the CHMP decided that Holoclar's benefits are greater than its risks and recommended that it be approved for use in the EU. The benefit / risk conclusion of Holoclar is based on the results of two retrospective studies (conducted with the use of past clinical data). The company will make available additional data from a prospective study (which records outcomes over the course of the study). Therefore, Holoclar has been given a "conditional approval". This means that more information on the medicine will be available in the future, which the company is required to provide. Each year the European Medicines Agency will review the new information available and this summary will be updated accordingly.

What information is still awaited for Holoclar?

As a "conditional approval has been given for Holoclar, the company that markets Holoclar will provide further data on the medicine. In particular, the company will make data on the risks and benefits of Holoclar available from a prospective clinical study."

What measures are being taken to ensure the safe and effective use of Holoclar?

A risk management plan has been developed to ensure that Holoclar is used as safely as possible. Based on this plan, safety information has been added to the summary of product characteristics and package leaflet for Holoclar, including the appropriate precautions to be followed by healthcare professionals and patients. In addition, the company that makes Holoclar will provide healthcare professionals with educational materials on the safe use of the medicine, including information on patient selection and monitoring at the end of the study as well as on reporting side effects. Patient information materials will also be provided. candidates for treatment Further information can be found in the summary of the risk management plan

More information about Holoclar

On February 17, 2015, the European Commission granted a "Marketing Authorization" for Holoclar, valid throughout the European Union. For more information on Holoclar therapy, read the package leaflet (included with the EPAR) or consult your doctor or the pharmacist. The summary of the opinion of the Committee for Orphan Medicinal Products for Holoclar is available on the Agency's website: ema.Europa.eu/Find medicine / Human medicines / Rare disease designation. Last update of this summary: 02-2015.

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