The new top 10 most expensive drugs on the planet

BioMarin’s decision last night to set the wholesale acquisition cost of its new ultra rare disease drug Brineura for CLN2 at $702,000 — with a significantly lower net amount — creates a new entry in the list of the 10 most expensive therapies in the world. With uniQure’s gene therapy Glybera unused and heading for the shelves, we also decided to drop that, leaving Horizon in the top slot with its WAC price for Horizon’s Ravicti, at *$793,632, according to the original lineup from Reinsurance Group of America. Brineura is the new number 2. We’ll also note that you could argue for a place on this list for Biogen’s Spinraza, which starts with a $750,000 price for its first year, but then drops by half, falling below the new top 10.


Wholesale Acquisition Costs (List Price)


1. Ravicti $793,632

Company: Horizon Pharma
Category: Rare disease – orphan drug
(glycerol phenylbutyrate)

Approved initially for Hyperion in 2013 as a treatment for urea cycle disorders, Horizon saw the potential and acquired the company for about $1.1 billion, boosting its portfolio of rare disease drugs. (*This is the same price cited by AHIP in their pricing report, which cites WAC and AWP pricing data published in the Federal Supply Schedule and the REDBOOK, as of Sept. 30, 2015. A spokesman for Horizon disputes this number, saying the WAC is closer to $560,000.)

2. Brineura $702,000

Company: BioMarin
Category: Rare disease – orphan drug
(cerliponase alfa)

The drug treats ultra rare cases — 20 new diagnoses a year — of Neuronal Ceroid Lipofuscinosis Type 2 disease (CLN2), a form of Batten disease. The disease progresses rapidly, with recurrent seizures and vision loss occurring as most victims are quickly left unable to walk by the age of six before eventually killing them. The drug was tested in only 22 patients in an abbreviated study, with a comparison on the natural history of several dozen others.

3. Lumizyme $626,400

Company: Sanofi – Genzyme
Category: Rare disease – orphan drug
(alglucosidase alfa)

This drug for Pompe’s disease helped make the rare disease field popular in biopharma. Approved in 2010, Sanofi liked the profile at Genzyme and bought out the company for about $20 billion a year later. The FDA handed out their orphan drug designation for this drug in 1997.

4. Carbaglu $585,408

Company: Recordati
Category: Rare disease – orphan drug
(carglumic acid)

This drug treats rare cases of N-acetylglutamate synthase deficiency. Also approved in 2010, there are only a handful of cases of NAGs disease every year. The condition is characterized by hyperammonemia, encephalopathy, and respiratory alkalosis and has frequently led to the swift deaths of newborns.

5. Actimmune  $572,292

Company: Horizon
Category: Rare disease – orphan drug
(Interferon gamma 1-b)

Approved for severe, malignant osteopetrosis and chronic granulomatous disease, rare genetic diseases, Actimmune noted last last year that the drug failed a Phase III for Friedreich’s ataxia. Horizon obtained a separate orphan drug designation for the drug when it decided to mount the study.

6. Soliris $542,640

Company: Alexion
Category: Rare diseases – orphan drug

This is the drug that built Alexion, and its success has helped inspire some new rivals to see if they can do it one better in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. It’s also under review now for refractory generalized myasthenia gravis, which earned the company another orphan drug designation.

7. Alprolix $503,880

Company: Bioverativ
Category: Rare disease – orphan drug
(Coagulation Factor IX [Recombinant], Fc Fusion Protein)

This long-acting hemophilia B medicine was recently spun out — along with Eolctate — from Biogen and Sobi into Bioverativ, which started out life with hundreds of millions in cash and plans to build a rare disease drug pipeline. Plenty of new drugs are in the works now that could revolutionize this field.

8. Idelvion $500,000

Company: CSL Behring
Category: Rare disease — orphan drug
(albutrepenonacog alfa)

Approved last year at the FDA, Idelvion is another long-acting hemophilia B drug that is the first in its class to include the blood protein albumin. The drug is designed to replace Factor IX, a naturally occurring clotting factor missing in hemophilia B patients.

9. Naglazyme $485,747

Company: Biomarin
Category: Rare disease – orphan drug

Naglazyme was originally approved back in 2005 for mucopolysaccharidosis type VI (MPS VI). Only a few dozen patients are prescribed this enzyme replacement drug in the US every year.

10. Folotyn $450,540

Company: Spectrum Pharmaceuticals
Category: Rare disease – orphan drug

This drug first began attracting attention for its price back in 2009, when it was priced at $30,000 a month after being approved to treat rare cases of peripheral T-cell lymphoma. Allos, which developed the drug, defended the price as in line with other drugs for rare cancers. Spectrum bought out Allos in 2012.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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