Feasibility Study for Bedside Production of Recombinant Human Acid α-Glucosidase: Technical and Financial Considerations

Author(s): Mohammed H. Aldosari*, Marcel den Hartog, Hubertina Ganizada, Martijn J.W. Evers, Enrico Mastrobattista, Huub Schellekens

Journal Name: Current Pharmaceutical Biotechnology

Volume 21 , Issue 6 , 2020

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Graphical Abstract:


Objective: The high cost of orphan drugs limits their access by many patients, especially in low- and middle-income countries. Many orphan drugs are off-patent without alternative generic or biosimilar versions available. Production of these drugs at the point-of-care, when feasible, could be a cost-effective alternative.

Methods: The financial feasibility of this approach was estimated by setting up a small-scale production of recombinant human acid alpha-glucosidase (rhGAA). The commercial version of rhGAA is Myozyme™, and Lumizyme™ in the United States, which is used to treat Pompe disease. The rhGAA was produced in CHO-K1 mammalian cells and purified using multiple purification steps to obtain a protein profile comparable to Myozyme™.

Results: The established small-scale production of rhGAA was used to obtain a realistic cost estimation for the magistral production of this biological drug. The treatment cost of rhGAA using bedside production was estimated at $3,484/gram, which is 71% lower than the commercial price of Myozyme ™.

Conclusion: This study shows that bedside production might be a cost-effective approach to increase the access of patients to particular life-saving drugs.

Keywords: Affordability, bedside production, orphan drug, Pompe disease, rare disease, rhGAA.

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Year: 2020
Page: [467 - 479]
Pages: 13
DOI: 10.2174/1389201021666200217113049
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