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

Mohammed H Aldosari; Marcel den Hartog; Hubertina Ganizada; Martijn J W Evers; Enrico Mastrobattista; Huub Schellekens

doi:10.2174/1389201021666200217113049

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

Mohammed H Aldosari
, Marcel den Hartog
, Hubertina Ganizada
, Martijn J W Evers
, Enrico Mastrobattista
, Huub Schellekens

Research output: Contribution to journal › Article › Academic › peer-review

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.

Original language	English
Pages (from-to)	467-479
Number of pages	13
Journal	Current Pharmaceutical Biotechnology
Volume	21
Issue number	6
DOIs	https://doi.org/10.2174/1389201021666200217113049
Publication status	Published - 2020

Keywords

Affordability
bedside production
orphan drug
Pompe disease
rare disease
rhGAA

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.2174/1389201021666200217113049

Cite this

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title = "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{\texttrademark}, and Lumizyme{\texttrademark} 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{\texttrademark}.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 {\texttrademark}.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",

author = "Aldosari, \{Mohammed H\} and \{den Hartog\}, Marcel and Hubertina Ganizada and Evers, \{Martijn J W\} and Enrico Mastrobattista and Huub Schellekens",

note = "Copyright{\textcopyright} Bentham Science Publishers; For any queries, please email at [email protected].",

year = "2020",

doi = "10.2174/1389201021666200217113049",

language = "English",

volume = "21",

pages = "467--479",

journal = "Current Pharmaceutical Biotechnology",

issn = "1389-2010",

publisher = "Bentham Science Publishers",

number = "6",

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T1 - Feasibility Study for Bedside Production of Recombinant Human Acid α-Glucosidase

T2 - Technical and Financial Considerations

AU - Aldosari, Mohammed H

AU - den Hartog, Marcel

AU - Ganizada, Hubertina

AU - Evers, Martijn J W

AU - Mastrobattista, Enrico

AU - Schellekens, Huub

PY - 2020

Y1 - 2020

N2 - 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.

AB - 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.

KW - Affordability

KW - bedside production

KW - orphan drug

KW - Pompe disease

KW - rare disease

KW - rhGAA

U2 - 10.2174/1389201021666200217113049

DO - 10.2174/1389201021666200217113049

M3 - Article

C2 - 32065100

SN - 1389-2010

VL - 21

SP - 467

EP - 479

JO - Current Pharmaceutical Biotechnology

JF - Current Pharmaceutical Biotechnology

IS - 6

ER -

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

Abstract

Keywords

UN SDGs

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