Huntington’s Disease: The New Gene Therapy That Sufferers Cannot Afford

May 16, 2016

(The Guardian) – Advanced therapeutics for rare, so-called orphan diseases will be expensive. An extreme example is the price of Glybera, developed by the Dutch company Uniqure to combat a rare disease that causes pancreatitis, which was set at $1m per patient. Ionis-HTTRx will not be as expensive as Glybera – nowhere near – but it will still be so expensive that only governments and insurance companies will be able to afford it. Glybera is administered only once, whereas Ionis-HTTRx would be taken many times, meaning the drug company could recoup costs and make profit over a patient’s lifetime rather than in one lump sum.