Pharmacoeconomic review report. Cysteamine delayed-release capsules (Procysbi), Horizon Pharma Ireland Ltd.
Cysteamine (Procysbi), a delayed-release formulation containing 25 mg or 75 mg of cysteamine bitartrate (referred to as delayed-release cysteamine) was approved by Health Canada for the treatment of patients with nephropathic cystinosis. Cystinosis is a recessive autosomal genetic ultra-rare disease...
Saved in:
| Online Access: |
http://www.ncbi.nlm.nih.gov/books/NBK534498/ Full text |
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| Format: | Electronic eBook |
| Language: | English |
| Published: |
Ottawa (ON) :
Canadian Agency for Drugs and Technologies in Health,
2018
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| Series: | Common drug review clinical review report.
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| Subjects: |
| Summary: | Cysteamine (Procysbi), a delayed-release formulation containing 25 mg or 75 mg of cysteamine bitartrate (referred to as delayed-release cysteamine) was approved by Health Canada for the treatment of patients with nephropathic cystinosis. Cystinosis is a recessive autosomal genetic ultra-rare disease affecting 1 in 100,000 to 200,000 live births in Europe (1 in 62,500 in Saguenay-Lac-St-Jean, Quebec), characterized by renal and extrarenal (i.e., diabetes, osteopenia, muscular weakness and wasting, growth retardation, etc.) complications. Cysteamine acts through preventing or delaying the occurrence of renal and extrarenal complications and hence prolonging life expectancy. The recommended dose of delayed-release cysteamine is 1.3 g/m2 daily to be given in two equal doses per day. At the manufacturer's submitted price ($11.30 per 25 mg capsule, $33.89 per 75 mg capsule), the daily dose cost will vary from $372.90 in a two-year-old child (based on a body surface area of 0.63 m2) to $881.40 in adults (based on a body surface area of 1.50 m2), or $136,000 to $321,000 per patient annually. |
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| Item Description: | "Indication: For the treatment of nephropathic cystinosis." "Version: Final." |
| Physical Description: | 1 online resource (1 PDF file (33 pages)). |
| Bibliography: | Includes bibliographical references. |
| Source of Description, Etc. Note: | Description based on online resource; title from PDF title page (viewed March 8, 2019). |
| Funding Information Note: | Funding: CADTH receives funding from Canada's federal, provincial, and territorial governments, with the exception of Quebec. |
