EMA Drafts Opinion on Cell Therapy Module From EBMT Registry

Regulatory NewsRegulatory News | 29 June 2018 |  By 

The European Medicines Agency (EMA) on Friday released for consultation a draft qualification opinion on cellular therapy related to the European Society for Blood & Marrow Transplantation (EBMT) registry.

The 50-page report describes instances in which the cellular therapy module of the EBMT registry is deemed by EMA’s Committee for Medicinal products for Human Use (CHMP) as an appropriate data source for post-authorization studies to support regulatory decision making concerning CAR-T cell therapy for hematological malignancies, as well as CHMP’s response to the questions posed by EBMT.

CHMP said it “considers that the current status of the cellular therapy module of the EBMT registry (coverage, core dataset, governance, quality assurance approaches, and completeness of core 77 variables), may allow its use as a data source for regulatory purposes in the context of the following  studies concerning CAR-T cell therapies authorised for haematological malignancies”:
  • Drug utilization studies for total recorded population and by subgroup such as age, gender, center, etc.
  • Drug efficacy/effectiveness studies
  • Drug safety evaluation
“The cellular therapy module of the EBMT registry could be used as a tool to collect CAR-T cell therapy safety data with a particular focus on early and late stage a) adverse events of special interest (i.e. neurological events (incl. cerebral oedema), cytokine release syndrome (CRS)/macrophage activation syndrome (MAS), hematopoietic cytopaenias, tumour lysis syndrome (TLS), certain infections, depletion of normal B-cells / a-/hypo-gammaglobulinemia, new malignancies), b) drug-related (grade 3-4) adverse events (skin; respiratory, cardiovascular, hepatic, renal, gastrointestinal, other system events),” the draft says.

Other sections discuss individual study considerations, further recommendations for enhancement and 11 questions and answers with CHMP responses, as well as questions posed to the agency and with responses from EBMT.

CHMP Recommendations

Also on Friday, CHMP recommended nine medicines for approval, including the first CAR-T cell therapies in the EU: Kymriah(tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), which are advanced therapy medicinal products (ATMPs) intended for the treatment of certain blood cancers.

Kymriah and Yescarta are both already approved in the US and are the first medicines supported through EMA’s PRIority MEdicines (PRIME)scheme to receive a positive opinion from CHMP.

Draft qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 25-28 June 2018


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