IPQ Monthly Update, March 2015

The Monthly Update for March is focused on the daunting manufacturing and control challenges that industry and regulators are facing in guiding the expanding array of cell and gene therapy (CGT) products now under development to commercialization. While it is clear that these dramatically effective treatments will play an increasing role in patient care and in biopharma portfolios, they are pushing up against the boundaries of the existing CMC regulatory framework and there are a lot of issues that remain to be resolved.

The search for a risk-based framework that would provide the needed flexibility is heightened in that several of these therapies are drawing a breakthrough review status. The challenges of speeding up the CMC development process are daunting enough for conventional biotech products, but only expand for the CGTs, where there aren’t even established CMC pathways to work with.

While speeding up the complex review process may not be realistic, what is at issue is heightened sponsor/agency knowledge sharing to untangle where the quality/clinical connections lie and how to manage the risks as early in development as possible so that commercialization can become a reality.

The March Monthly Update focuses on the dialogue that has been intensifying between biopharma and regulators at recent public forums on these challenges and the approaches that are emerging to address them. The series of five stories IPQ developed during the month closely interconnect and are cross-linked in the issue.

Included are a pair of stories that explore the international dialogue on the regulatory issues the new therapies pose, what viable approaches are being developed to address them, and where harmonization opportunities lie.

The first of the international stories focuses on the insights of a top European CGT regulator on Europe’s perspective, experience and evolving policies. The second brings in the CGT regulatory developments in Japan and elsewhere in Asia and the dialogue on the divergences and convergences of the approaches taking shape globally.

The US component begins with an in-depth review of a very informative presentation by a leader of the Novartis program on the company’s advancing efforts to adapt biotech manufacturing and control technology and systems to make CGT commercialization a reality.

Stories follow on the especially challenging but critical task of developing potency assays for the advanced therapies.

The first of the two reviews insights at a recent forum from: ● a leading regulator at FDA’s CBER on the agency’s latest thinking about the issues at hand, and ● experts from three companies that are wrestling with developing viable CGT potency bioassays on what they are learning. The second story moves to the probing panel discussion that unfolded at the forum on the key potency assay challenges and where promising strategies lie.

Also provided in the Monthly Update are two dozen briefs on breaking developments in the US, European and international arenas – with links to the related documents and IPQ coverage – along with a review of the drug GMP warning letters and recalls announced by FDA during March.

To access the Monthly Update, subscribers please, click here.