Official encourages ATMP sponsors to consult toolbox guidance to avoid PRIME derailments

Regulatory NewsRegulatory News | 16 June 2022 |  By 

An EU official advised manufacturers of advanced therapy medicinal products (ATMPs) to consult the European Medicines Agency’s (EMA) recently issued “toolbox” guidance to avoid having products rejected from being eligible for accelerated review under the PRIME program for quality-related deficiencies.
Barbara Bonamassa, a reviewer with the Italian Medicines Agency (AIFA), offered an update on some of the EU’s ongoing activities to expedite the development of ATMP therapies at the California Separation Science Society’s (CASSS) meeting on 8 June on cell and gene therapies. The meeting was held both in-person in Arlington, VA and virtually.
Bonamassa is also a member of EMA’s Committee for Advanced Therapies and a quality assessor for biotherapeutic products for the World Health Organization’s Prequalification Team.
EMA launched its PRIME (PRIority MEdicines) scheme in March 2016 to expedite the development of new therapies that address unmet medical needs. (RELATED: EMA Looks to Accelerate New Therapies Targeting Unmet Need, Regulatory Focus 7 March 2016)
Bonamassa noted EMA’s recent report on the first five years of the PRIME scheme, which contains “important and relevant information about ATMPs.” (RELATED: EMA lauds PRIME priority meds and outlines potential revisions, Regulatory Focus 8 March 2022)
According to the report, “Although ATMPs account for approximately 27% of the requests received for PRIME eligibility, they present the highest success rate (corresponding to 46% of all PRIME products). This is because the submitted requests generally combined a potential to address an unmet medical need with a usually very specific mechanism of action and strong demonstration of proof of concept.”
Yet Bonamassa notes that that “quality related deficiencies” often derail these products from formal acceptance into the program and gaining accelerated review status.
Bonamassa said EMA issued toolbox guidance in April 2022 to avoid having their packages rejected for these deficiencies. The guidance addressees process validation protocols; continuous process verification; approaches related to GMP compliance’ alignment of quality assessments and GMP inspections of biological starting materials; stability; and establishing product comparability. (RELATED: New toolbox available for EMA's PRIME designees, Regulatory Focus 2 February 2021)
“I strongly encourage you to read the toolbox guidance for reading it you will see that the guidance is not meant to reduce the quality requirements in applications,” she said.
In other areas, she said the EMA’s Innovation Task Force (ITF) is also working to expedite the development of ATMPs. The task force includes a “cross sectional group which is meant to focus on the emerging therapies and technologies.”
The group engages in early dialogue with applicants to identify scientific, legal and regulatory issues relating to their applications. Bonamassa said that interest in these meetings has grown: the number of ITF requests for meetings has doubled over the last three years, and the number of meetings has increased as well.
Bonamassa added that “this tool is freely used by applicants in the early stage of development even before a formal request for scientific advice is requested.”
EMA has also established a new expert group to help expedite the development of ATMPs which aims to and develop innovative chemistry, manufacturing, and controls (CMC) approaches for ATMPs and other types of chemical or biological products.
CASSS meeting on cell and gene therapies


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