MHRA drafts mRNA cancer immunotherapy guidance

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) is seeking input on its draft guidance for developing individualized messenger ribonucleic acid (mRNA) cancer immunotherapies. The agency said that research into the treatments is advancing rapidly, and it wants to ensure its premarket pathways are prepared to allow such products on the market.

Since the technology was successfully used to develop COVID-19 vaccines, drugmakers have sought to apply it in new therapeutic areas, with oncology being a major focus.

“Individualized cancer immunotherapies, while still being tested in clinical trials, are a very exciting development in our hunt to find new and better ways to treat cancer, which is a leading cause of death worldwide," said Julian Beach, MHRA executive director of healthcare quality and access. "Because these treatments are tailored to an individual’s tumor, they pose unique scientific questions on how they should be regulated.”

Individualized mRNA cancer immunotherapies are being developed to use mRNA as a messenger to train a patient’s own cells to make specific proteins to fight cancer.

MHRA said its proposed guidance only addresses mRNA cancer immunotherapies that use lipid nanoparticle delivery systems. More specifically, it is intended to cover cancer immunotherapies that contain mRNA as the active substance encapsulated in lipid nanoparticles that deliver the vaccine.

Regulators said they will update the guidance as they gain experience in other technologies, such as peptides, non-integrative DNA, and polymer delivery systems.

MHRA noted that cancer immunotherapies are often called cancer vaccines. However, based on definitions in the Human Medicines Regulations (HMRs), individualized mRNA cancer immunotherapies do not meet the definition of vaccines, so instead, the agency will refrain from using the term. The therapies in these cases are tailored to a patient's unique tumor neoantigen profile.

“The MHRA envisages that in specific circumstances, an individualized medicine could be issued with a marketing authorization (MA) under the HMRs, even where there is a variable component that is tailored to an individual patient’s characteristics,” said MHRA. “This means that a single MA could cover use across the target population defined in the indication.”

MHRA said the production requirements, strength, pharmaceutical form, and method of administration would otherwise be the same between different patient-specific batches. However, while the agency could be flexible on the specifics, the manufacturer would need to follow predefined processes and controls to ensure the product is safe and effective, and the product’s indication would need to be backed by relevant clinical and non-clinical data.

The draft guidance delves into product design, manufacturing, and clinical and non-clinical considerations. It also contains a section on post-authorization requirements that includes risk management plans and a section on how to inform patients and healthcare professionals about the risk-benefit of mRNA cancer immunotherapies.

Public health officials encouraged stakeholders to comment on the proposed guidance and emphasized that they do not want to hold back mRNA cancer immunotherapies from coming to market due to a lack of guidance.

“Personalized immunotherapies could revolutionize our approach by helping patients fight cancer cells in their bodies,” said Public Health Minister Andrew Gwynne. “As government ramps up the use of groundbreaking technologies and medicines across the board, this guidance will be fundamental to achieving our goal of moving from sickness to prevention.”

Stakeholders can comment on the guidance until 31 March on MHRA’s website here.

Guidance announcement

MHRA drafts mRNA cancer immunotherapy guidance

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