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

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.

journal_name

Curr Pharm Biotechnol

authors

Aldosari MH,den Hartog M,Ganizada H,Evers MJW,Mastrobattista E,Schellekens H

doi

10.2174/1389201021666200217113049

subject

Has Abstract

pub_date

2020-01-01 00:00:00

pages

467-479

issue

6

eissn

1389-2010

issn

1873-4316

pii

CPB-EPUB-104561

journal_volume

21

pub_type

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