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GLYBERA (alipogene tiparvovec), gene therapy
RARE DISEASE - New medicinal product
Opinions on drugs -
Posted on
Jan 28 2016
Reason for request
Inclusion
Insufficient clinical benefit because of the moderate, heterogeneous and unsustained effect on the blood triglyceride level and the prevention of pancreatitis and because of the uncertainties about its short- and medium-term safety
- GLYBERA has Marketing Authorisation in the treatment of adult patients with familial lipoprotein lipase (LPL) deficiency who have severe or multiple pancreatic crises despite a low-fat diet.
- A moderate effect on triglycerides and on episodes of pancreatitis has been observed but this effect was not sustained in the medium- and long-term (return to the baseline triglyceride level 1 year after the injection) and inter-patient heterogeneity of the treatment response.
- Uncertainties about the short- and medium-term safety of this gene therapy, which cannot be re-administered because of its action mechanism, remain.
Clinical Benefit
| Insufficient |
- |
Therapeutic use
|
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