CHMP: AMT's Glybera, Europe's first gene therapy, still not approvable
This article was originally published in Scrip
The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has maintained its earlier opinion on Amsterdam Molecular Therapeutics' Glybera (alipogene tiparvovec), saying it is still not approvable at this time.
AMT's share price took a hammering on the day of the news, dropping from €0.80 to a low of €0.25, a 69% fall. By the close of trade there was a slight recovery to €0.38, which, nevertheless, represented a 53% decline.
The company had requested a re-examination of the marketing authorisation approval application for the product for a rare genetic disease, lipoprotein lipase deficiency (LPDL), after being turned down by the EMA's scientific committee earlier this year (scripintelligence.com, 8 July 2011 & 24 June 2011).
The company highlighted that the latest CHMP decision was in contrast to the positive recommendation of the committee's Scientific Advisory Group (SAG; the expert panel specifically selected to evaluate clinical results and the science of the product) as well as its Committee for Advanced Therapies (CAT), which provides guidance on advanced therapeutics such as gene and cell therapy.
Following extensive review and analysis of the data, these expert panels had advised the CHMP that Glybera should be approved now under exceptional circumstances, AMT said. SAG and the CAT had concluded that data from three Glybera clinical trials demonstrated meaningful evidence of clinical efficacy, without any major safety concerns but the CHMP has not adopted the same opinion.
"While AMT carefully reviews the grounds of the CHMP refusal, the company will focus on strategic advancement of other promising projects in its development pipeline," the company stated.
It added: "We are encouraged by the validation provided by the CHMP Rapporteurs, Scientific Advisory Group and the Committee for Advanced Therapies regarding our gene therapy platform, indicating that safety is not an issue. We are evaluating thoroughly and expediently the next steps available to the company and will provide an update on the future developments of our validated gene therapy pipeline."
Had the product been successfully approved, it would have been the first gene therapy to be marketed in Europe and also a much needed treatment option for patients with no other treatment options.
Even though AMT's CEO, Jörn Aldag, has remained 'convinced and confident' of the approval prospects for Glybera the regulatory path for the drug has been far from smooth. AMT's initial filing of Glybera was on 23 December 2009 and resulted in a list of questions received by AMT in May 2010. After two meetings with the EMA in July 2010, AMT stated that it would not be required to conduct more clinical trials and submitted their response to the Agency's questions in November 2010. On day 180 in the regulatory process, which would have been at the end of January 2010, the EMA sent another list of questions to AMT. As late as May this year the AMT CEO stated that their interactions with the EMA were positive and that they were looking forward to a decision in mid-2011. That decision of non-approval came five weeks later in part, due to insufficient clinical benefit as a result of low patient numbers (scripintelligence.com, 11 July 2011).