EMA updates: Zynteglo safety review, clinical trial information system and more

Regulatory NewsRegulatory News | 12 March 2021 |  By 

The European Medicines Agency’s Management Board and Pharmacovigilance Risk Assessment Committee (PRAC) on Friday released lengthy updates on a range of issues from the rollout of the EU IT systems required by the Clinical Trial Regulation and safety reviews of several medicines.
Management Board updates
After its meeting on Thursday, EMA’s Management Board stressed the need for specialized experts and resources at the national level to support EMA’s work to review and monitor vaccines and therapeutics for COVID-19 “to ensure that EMA can continue to conduct its assessments using the best possible EU expertise and within the shortest possible timeframes.”
The Management Board said it was reassured that the agency has shored up its cybersecurity defenses after a cyberattack last year resulted in the unauthorized access to documents related to COVID-19 vaccine submissions from Pfizer/BioNTech and Moderna.
The Management Board also provided an update on EMA’s long-delayed clinical trial information system (CTIS), which is now expected to go live by 31 January 2022. According to the Management Board, the first phase of the independent audit of the system has been completed and preparations for the second phase of the audit are underway.
“The requirements for a minimum viable product at the time of go-live have been prioritised by the group of experts mandated by the CTIS governance. On the basis of this prioritisation, a development plan for go-live and subsequent post-go-live periods has been prepared,” EMA writes, noting that the plan includes very limited additional functionality for the system.
PRAC updates
Following the PRAC meeting earlier this week, EMA divulged that the committee is undertaking a safety review of bluebird bio’s beta thalassemia gene therapy Zynteglo (betibeglogene autotemcel) and provided several updates related to COVID-19 vaccines and other medicines.
The safety review of Zynteglo comes after the company halted clinical trials of another gene therapy that uses the same lentiviral vector after a participant in the trial was diagnosed with acute myeloid leukemia (AML).
However, bluebird this week said that its own analyses found that the case of acute myeloid leukemia (AML) reported in the trial was unlikely to be related to the lentiviral vector.
“So far, no cases of leukaemia have been reported with Zynteglo itself,” EMA said, noting that bluebird has halted the supply of Zynteglo while the review is being conducted. “PRAC will now examine the evidence thoroughly at the EU level, working closely with experts from EMA’s Committee for Advanced Therapies (CAT), which is responsible for assessing this type of medicine, and decide on any relevant regulatory action for Zynteglo.”
Along with a statement on its review of thromboembolic events after vaccinations with AstraZeneca’s COVID-19 vaccine, EMA also said that PRAC concluded its assessment of cases of anaphylaxis occurring after administration with the vaccine. (RELATED: EMA offers reassurance on AZ vaccine, while some European authorities pause, Regulatory Focus 11 March 2021).
“PRAC has recommended an update to the product information to include anaphylaxis and hypersensitivity (allergic reactions) as side effects in section 4.8, with an unknown frequency, and to update the existing warning to reflect that cases of anaphylaxis have been reported,” EMA writes, noting that there were 41 reports of possible anaphylaxis among 5 million vaccinations in the UK. “After careful review of the data, PRAC considered that a link to the vaccine was likely in at least some of the cases.”
EMA said that PRAC is launching a safety review of all three COVID-19 vaccines in widespread use in the EU from Pfizer/BioNTech, Moderna and AstraZeneca to investigate reports of immune thrombocytopenia.
While several cases of the bleeding disorder have been reported in the EudraVigilance database, EMA said, “It is not yet clear whether there is a causal association between vaccination and the reports of immune thrombocytopenia.”
The committee has also requested that Pfizer and BioNTech review reports of localized swelling in people who have had dermal fillers and received the companies’ COVID-19 vaccine Comirnaty. The agency notes that skin reactions associated with dermal fillers were first reported by the Norwegian Medicines Agency and a limited number of cases have been reported to EudraVigilance.
Additionally, PRAC concluded that the benefits of ifosfamide solutions for infusion continue to outweigh their risks in the treatment of certain solid tumors and blood cancers after recent studies suggested that ifosfamide solutions pose an increased risk for encephalopathy compared to the drug in its powder form. However, EMA said there was not enough data to confirm or deny the increased risk and recommended updated the existing warnings to include the latest information on the side effect.
EMA Management Board, PRAC


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Tags: EMA, EU

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