Δευτέρα, 8 Μαΐου 2017

MOST EXPENSIVE DRUGS

Leading that pack at number 1 is glycerol phenylbutyrate (Ravicti, Horizon Pharma), an orphan drug approved by the US Food and Drug Administration (FDA) in 2013 for the long-term management of urea cycle disorders, which are estimated to affect 1 in 8500 births. The list price per year for Ravicti is about $793,000, according to the analysis compiled by Endpoints News. But a spokesperson for Horizon Pharma told Medscape Medical News that the accurate list price for Ravicti is approximately $556,000 per year, which would put it lower on the list.
Taking the number 2 spot on the list is orphan drug cerliponase alfa (Brineura, BioMarin International Ltd), approved last month by the FDA for the treatment of infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease. The drug will cost an estimated $702,000 a year. About 20 new cases of CLN2 are diagnosed each year.
Coming in at number 3 is alglucosidase alfa (Lumizyme, Sanofi-Genzyme), approvedin 2010 to treat late-onset Pompe disease in children aged 8 years and older. It has a list price of about $626,400 per year, according to the analysis.
Orphan drug carglumic acid (Carbaglu, Recordati), for treating the rare genetic disorder N-acetylglutamate synthase deficiency, which causes hyperammonemia, holds the number 4 spot on the list of most expensive drugs. The drug was approvedin 2010 and has a list price of about $585,400 per year.
Number 5 on the list is interferon gamma 1-b (Actimmune, Horizon Pharma), approved to decrease the number and severity of infections in patients with chronic granulomatous disease and to delay the progression of severe, malignant osteopetrosis. The list price tops $572,000 annually, according to the analysis. However, Horizon Pharma said the accurate list price for Actimmune is approximately $480,000/year.

Rounding out the top 10 most expensive drugs are the following

6. Eculizumab (Soliris, Alexion Pharmaceuticals), approved for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. It has an estimated list price of $542,640 per year.
7. Coagulation factor IX recombinant Fc fusion protein (Alprolix, Bioverativ), the first long-acting hemophilia B drug approved by the FDA for use in children and adults with hemophilia B. It has a list price of about $503,800 per year.
8. Albutrepenonacog alfa (Idelvion, CSL Behring), another long-acting hemophilia B drug. Approved in 2016, Idelvion combines albumin with factor IX to reduce injection frequency. It has an annual price tag of $500,000.
9. Galsulfase (Naglazyme, Biomarin Phamaceutical), approved in 2005 to improve walking and stair-climbing capacity of patients with mucopolysaccharidosis VI. It lists for around $485,700 per year.
10. Pralatrexate (Folotyn, Spectrum Pharmaceuticals) was approved in 2009 to treat relapsed or refractory peripheral T-cell lymphoma. The relatively rare and often aggressive type of non-Hodgkin's lymphoma occurs in fewer than 9500 US patients annually. Folotyn has a list price of around $450,500.

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