Biomarin and the $702,000 question


Biomarin won a widely anticipated, although somewhat delayed, US approval for Brineura for a rare form of Batten disease yesterday, gaining in the process a useful and potentially valuable rare paediatric disease priority review voucher. However, investors pushed down its stock by 4% in the after-market seemingly on concerns that its pricing strategy for this ultra-rare condition might draw flak in the current political environment.

The company plans to launch the drug in early June with a wholesale price of $27,000 per 300mg carton, which, at a carton every two weeks, is equivalent to $702,000 per year. The group is doing what it can to cut this, saying the net price should average $486,000 per year after discounts, but this still puts it in third place in among the most expensive drugs (see table).

At $486,000 – assuming Biomarin can hit this goal – Brineura would be broadly similar with the same company’s Naglazyme and around 20% below the real-world price for Alexion’s Soliris, the benchmark for all ultra-rare drugs.

Top 10 most expensive drugs
Product Company USA Cost per Patient per Year ($)*
Soliris Alexion Pharmaceuticals 599,842
Naglazyme Biomarin Pharmaceutical 492,213
Brineura Biomarin Pharmaceutical 486,000**
Eloctate Bioverativ 459,332
Cerezyme Sanofi 457,930
Gazyva Roche 449,984
Cinryze Shire 416,029
NovoSeven Novo Nordisk 369,245
Fabrazyme Sanofi 361,057
BeneFIX Pfizer 336,479
Note: all orphan drugs, *an estimate for the retail cost of a drug to a patient, for a given year, based on a 100% compliance to the treatment guidelines outlined in the FDA label; **Biomarin net price estimate
Source: EvaluatePharma

The CLN2 form of Batten disease is extremely rare with only around 20 children born with the condition each year in the US. A disease of the nervous system, symptoms include language delay, seizures and difficulty coordinating movements; affected children lose the ability to walk and talk at around six years of age, with death occurring between eight and 12.

Biomarin estimates the global incidence of CLN2 disease to be one in 200,000 and suggests there may be 1,200-1,600 children in the regions of the world where it operates with the condition, many of whom are undiagnosed.

Brineura’s launch in the EU is also likely later in the year, as the CHMP adopted a positive opinion last week and the European Commission is expected to render its final decision by the end of second quarter.

Biomarin has guided analysts to expect a relatively measured launch trajectory, with its initial focus on the identification of patients. Consensus forecasts suggest sales of $9m this year, rising to around $249m in 2022, according to EvaluatePharma. However, sales should continue to grow beyond that, with analyst Edward Nash of SunTrust Robinson Humphrey, for example, projecting sales of $344m in 2026.


The drug is the first enzyme replacement therapy to be directly administered to the cerebrospinal fluid by intraventricular administration. It was approved to slow the loss of ambulation in paediatric patients aged three years or older, based on a study of 23 children with CLN2 disease assessed over 96 weeks, compared with untreated patients from a natural history cohort.

Results in the study were good, with the motor functions of 21 of the 22 Brineura-treated patients not worsening. The data suggested that a Brineura-treated patient was 13 times less likely to decline than those in the historical control cohort.

Positive data and the emotive nature of the disorder will still not be enough for Biomarin to avoid pricing pushback, however – even with the discounts it promises.

To contact the writer of this story email Robin Davison in London at or follow @RobinDavison2 on Twitter

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