• DOI:10.1038/nbt0315-217
• Corpus ID: 205266596

$1-million price tag set for Glybera gene therapy

@article{Morrison20151millionPT,
  title={\$1-million price tag set for Glybera gene therapy},
  author={Chris Morrison},
  journal={Nature Biotechnology},
  year={2015},
  volume={33},
  pages={217-218}
}

• C. Morrison
• Published 6 March 2015
• Medicine
• Nature Biotechnology

an annuity model that spreads that payment over a number of years to lessen the costdensity burden on payers; and a pay-forperformance, risk-sharing model that tracks patient outcomes and rewards manufacturers for maintaining patients’ health over a period of time. Each of these schemes is likely to be used, depending on the specific attributes of any one therapy and specific negotiations between drug manufacturers and payers. Rapid progress in the clinic may speed up such decisions. Bluebird’s therapy, was granted FDA’s breakthrough therapy designation on February 2, and since the company’s December presentation at the hematology meeting, eight patients have been treated with the drug across two phase 1/2 clinical studies. Together, the two trials will enroll up to 22 patients with either beta-thalassemia major or severe sickle cell disease. BB305 treats those diseases by using a lentiviral vector to transfect a patient’s own hematopoietic stem cells ex vivo with a funcThe first gene therapy approved in the Western world is set to go on sale in Germany at a cost close to $1 million per treatment. The record-breaking price tag came to light in November 2014, when Amsterdam-based Uniqure and its marketing partner Chiesi, of Parma, Italy, filed a pricing dossier with German authorities to launch Glybera. A few weeks later, the focus on gene therapies sharpened further when Cambridge, Massachusetts–based Bluebird Bio presented striking early clinical data from four beta-thalassemia patients treated with its Lentiglobin BB305 gene therapy, at the American Society of Hematology meeting in San Francisco. Within three months, these patients had begun producing sufficient hemoglobin to reduce or eliminate the need for blood transfusions. Big pharma is taking notice; the most recent gene therapy deal signed in February, between Sanofi’s Genzyme unit and Third Rock Ventures’ Voyager Therapeutics, both in Cambridge, Massachusetts, is worth up to $845 million. But as gene therapies start to provide solutions for highly penetrant genetic diseases that had been intractable, the hand-wringing over their value and how government and private insurers around the globe will pay for them will likely begin. “The pricing of specialty medicines has now caught everyone’s attention,” says Troyen Brennan, chief medical officer for the pharmacy benefit manager CVS Caremark, based in Woonsocket, Rhode Island. The pricing debate, sparked by Gilead Science’s hepatitis C virus drug regimen Sovaldi (sofosbuvir; Nat. Biotechnol. 32, 501–502, 2014), is set to intensify with the advent of these potentially curative—and very expensive—therapies for certain rare genetic diseases. As the first and only approved gene therapy in Europe or the US, Glybera (alipogen tiparvovec) will likely become a benchmark product. Glybera was recommended for approval by the European Medicines Agency in 2012 (Nat. Biotechnol. 30, 1153, 2012), and Chiesi plans to launch the product in Europe in early 2015, though it has yet to receive a nod from the US Food and Drug Administration (FDA). Glybera is used to treat adults with lipoprotein lipase (LPL) deficiency, an orphan disease which results in abnormally large particles of fat in the blood and causes inflammation of the pancreas. The therapy consists of LPL, encoded by an adeno-associated virus vector, administered through a series of intramuscular injections. In addition to Germany, the company expects UK pricing March 1. So far, pricing discussions for gene therapy has remained “under the radar” for many payers in the US, says Brennan. “I’ve not seen the national health insurers talking much about this, but they will be sooner or later.” Glybera’s price depends on a patient’s weight, explained Uniqure CEO Jörn Aldag, at an investor meeting in early December. An average patient would need 19 vials of Glybera, he said, at a cost of nearly €44,000 (about $50,000) per vial. “Higher value to patients ... should actually command a higher price,” Aldag noted. Uniqure has six-year follow-up data showing a 50% reduction in pancreatitis in Glybera-treated patients, he said, and dividing that up-front price by six years “you get to a [per-year] price that’s actually lower than [typical] orphan drug pricing,” he said. Pricing gene therapies might follow one of three general schemes. A classic up-front, one-time payment, such that Chiesi and Uniqure are seeking for Glybera in Europe;