Four and a half years after making history and getting European Medicines Agency approval for the first gene therapy in a regulated market, uniqure NV is throwing in the towel and will not apply for the license to be renewed when its expires on 25 October.
The decision is not unexpected: only one patient has been treated with the commercial form of €1.1 million therapy since its approval. While the company hoped the European approval would pave the way to getting the nod from the FDA, the agency asked for two further trials, forcing uniqure to abandon its plan to market Glybera in the US.
“The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern,” Matthew Kapusta, chief executive officer of uniQure. “Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead.”
Although uniqure was blaming high exclusion criteria rather than cost, for the commercial failure of Glybera, the withdrawal sends a signal that Europe’s hard-up healthcare systems are not prepared to pay for innovative therapies being developed by its biotech companies. Glybera is only reimbursed in Germany and Italy.
After 14 years in development and a great deal of controversy, in October 2012 the European Commission granted a five-year marketing authorisation for Glybera, a treatment for a rare inherited disorder which prevents patients from digesting dietary fat.
Special terms attached to the authorisation required the company establish a global registry for the long-term surveillance of patients, conduct a post-approval clinical study, submit for annual regulatory reassessments and implement additional risk management procedures.
Uniqure said this meant it has to pay for significant infrastructure, including maintaining a commercial manufacturing facility, developing, developing and validating numerous assays and supporting regulatory interactions and inspections.
The axeing of Glybera will add to the pressure that is building in Europe for a new approach to reimbursement of expensive one-off cell and gene therapies. That would allow companies to get a return on investment without creating an unsustainable financial burden for healthcare systems.
Although the €1.1 million price tag of Glybera looks excessive, its effects are long-lasting. A one time, single-administration gene therapy, which introduces copies of natural LPL gene to produce functional LPL enzyme, provides a long-term therapeutic effect, currently documented up to six years from administration.
Comparable enzyme replacement therapies require continuous administration and cost €150,000 - €450,000 per year.
Amsterdam-based uniQure said it is talking to the European Medicines Agency (EMA) to discuss steps to wind down various Glybera-related activities and review plans for ongoing patient monitoring.