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Development of method for mobile aseptic preparation of advanced therapy medical products

European Statement

Production and Compounding

Why was it done?

The pharmacy received a request for reconstitution of an ATMP Luxturna. The pharmacy or hospital did not have a suitable aseptic facility that could perform the reconstitution in a way that allowed the ATMP to be administered to patients before the expiry after reconstitution (max. 4 hours).

What was done?

Development of a single use isolator for advanced therapy medical products (ATMP) or gene therapy drug preparation was undertaken. The single use isolator had to be mobile to enable pharmacy staff to preform reconstitution directly on ward or in OP-theatre. It had to comply with Health and Safety regulations and at the same time make it possible for pharmacy staff to use aseptic technique to be able to reconstitute ATMP.

How was it done?

A review of isolator technology was performed, and a suitable solution identified. The set-up was further developed in a team with representatives from pharmacy, the eye department at Rigshospitalet-Glostrup. A manufacturer of the equipment was selected, and development performed. The set-up for ATMP preparation was presented to The Danish Health and Safety (DHS) department responsible for handling this type of treatment. The approval was granted after a standard 90-day period. Pharmacy staff were trained in working with the set-up and a dry run made in the eye theatre with the full surgical team.

What has been achieved?

10 patients (19 eyes) have been treated, 4 male and 6 female patients with age span 12-39 years. After having performed the procedure twice there was enough routine to treat 2 patients per theatre day hence reducing the cost of preparation and increasing efficiency of the team.
The pharmacy and surgical team have established a great working relationship and now consider the set-up as routine.
In spring 2021 the set-up was approved by (DHS) for reconstitution of Zolgensma. On July 1 a pediatric patient was treated with Zolgensma using the method.

What next?

Plan to develop the method further and make it available for coming preparations of ATMPs and share knowledge of the method with other hospital pharmacy organizations.

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