uniQure’s Glybera becomes the first gene therapy approved in a regulated market


Source: Madprime http://bit.ly/RLmMqL

On October 11, 2012 we published an article entitled “Is gene therapy emerging from technological prematurity?” on the Biopharmconsortium Blog. The centerpiece of that article was the July 20, 2012 ruling by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) that recommended marketing uniQure’s Glybera. Glybera (alipogene tiparvovec) is a gene therapy for the ultra-rare genetic disease lipoprotein lipase deficiency (LPLD).

On November 2, 2012, the European Commission approved Glybera, which now becomes the first gene therapy approved in a regulated market. This was announced by uniQure, and covered by BioWorld Today and Reuters, among others.

Now that Glybera is approved in Europe, uniQure is exploring registration of Glybera in North America, and is developing its strategy for interaction with relevant regulators, especially the FDA. uniQure is aiming for a U.S. launch of Glybera in 2014.

According to uniQure, the commercial roll-out of Glybera will begin in the second half of 2013. uniQure estimates that there are 400 to 500 patients in Europe eligible to receive the therapy.

uniQure also says that the approval of Glybera validates the company’s  adeno-associated virus (AAV) vector-based gene therapy platform. In that connection, uniQure is planning to develop four other gene therapies that utilize its platform–treatments for hemophilia B, for acute intermittent porphyria, for Parkinson’s disease, and for Sanfilippo B, a rare liposomal storage disorder. These four gene therapy products have approval to enter clinical trials within the next nine months.

uniQure faces a short-term funding gap until revenues from Glybera start coming in. It is seeking to raise €20 million (US$26.7 million) in investment over the next five months.

uniQure’s ability to successfully commercialize Glybera depends on the pricing for the therapy allowed by payers. The company is now negotiating with payers to set prices. uniQure is basing its pricing for Glybera for the prices of enzyme replacement products for treating lysosomal storage disorders, such as those developed by Genzyme. For example, Genzyme’s Cerezyme (imiglucerase), a treatment for Gaucher’s disease, cost $200,000 per year in the United States in 2009. However, unlike Genzyme’s enzyme replacement therapies, Glybera, being a gene therapy, is a one-time treatment designed to restore a natural body function rather than providing short-term amelioration of a genetic disease.

According to the Reuters article, Glybera is expected to cost approximately €1.2 million ($1.6 million) per patient. This would be a new record for expensive modern therapeutics. Jörn Aldag, uniQure’s CEO, believes that the high price is justified by the long-term benefit provided by a gene therapy, as opposed to the classic protein replacement strategy in which the drug must be administered repeatedly for life.

Different European countries prefer different payment schemes for Glybera. Some favor charging an annual price for the therapy, while others prefer a single up-front charge, based on multiplying the annual cost of treating a similar disease (e.g., Gaucher’s disease) by the number of years Glybera is known to have an effect. Currently, that is five years.

For an in-depth discussion of the prospects for the gene therapy field, and the implications of the approval of Glybera for the future of gene therapy, see our October 11, 2012 article on this blog.


As the producers of this blog, and as consultants to the biotechnology and pharmaceutical industry, Haberman Associates would like to hear from you. If you are in a biotech or pharmaceutical company, and would like a 15-20-minute, no-obligation telephone discussion of issues raised by this or other blog articles, or an initial one-to-one consultation on an issue that is key to your company’s success, please contact us by phone or e-mail. We also welcome your comments on this or any other article on this blog.

Visited 1949 Times, 1 Visit today