Luxturna, a drug against hereditary retinal degeneration that can lead to total blindness, will be sold for $850,000 in the United States, making it one of the most expensive treatments in the world.
It is administered in a single dose, according to its manufacturer, the biotech company Spark Therapeutics, based in Philadelphia. In detail, it will cost $425,000 per eye, but the final price remains below the $1 million originally envisioned when it received approval from the U.S. Food and Drug Administration (FDA) in mid-December.
This announcement comes in the midst of a debate on the high cost of medicines in the United States, particularly the prices of so-called innovative treatments, and should not fail to provoke protests. In response to criticism, Spark has promised to reimburse patients if the treatment is not effective and is also considering asking health authorities to authorise payment in stages.
The biotech is based on an approach recently adopted by the Swiss laboratory Novartis when it began marketing Kymriah, which is supposed to treat a very aggressive form of leukemia in children and young adults. The Swiss laboratory is committed to returning the money to insurers and patients if this treatment, sold for USD 475,000, is ineffective.
Luxturna is considered to be the first American drug resulting from gene therapy, which consists of correcting a genetic anomaly by repairing the defective gene. About 1,000 cases of people suffering from hereditary retinal degeneration are currently registered in the United States and 10 to 20 new cases are expected per year over the next few years.
In addition to Luxturna, other drugs currently cost around $1 million, although they often require several doses. This is the case of Spinraza (Nusinersen in Europe), developed by Biogen and Ionis Pharmaceuticals against muscle atrophy, and Soliris, manufactured by Alexion Pharmaceuticals to treat a rare kidney disease. They both cost about $750,000.
The world's most expensive drug, however, is Glybera, the first gene drug sold for $1 million by the Dutch group UniQure to treat several genetic diseases.