Regulatory Round-up - March 2022

United Kingdom

Medicines and Healthcare products Regulatory Agency (MHRA)

Optimising the Market Access Approach - ILAP HTA Access Tool

The MHRA has updated information in relation to the ILAP HTA Access Tool accessible for Innovation Passport (IP) holders. This tool aims to promote understanding of the market access approaches that will optimise the route to patient access. This tool, which is delivered by the NICE Office for Market Access (OMA) in collaboration with the Scottish Medicines Consortium (SMC) and All Wales Therapeutics and Toxicology Centre (AWTTC), aims to provide a joined-up view from system stakeholders in England, Scotland and Wales.

Full details can be found on The Target Development Profile Toolkit webpage

British Pharmacopeia (MHRA)

Guidance on the application of vector copy number quantification for the ATMP community

On 23^rd March 2022, the British Pharmacopeia published its second non-mandatory, best practice guidance “The application of vector copy number quantification” in a series of guiandace documents aimed at supporiting the ATMP community. The guidance is split into two parts focused on part I: Adeno-associated virus, and part II: Lentivirus and Retrovirus, respectively. This follows on from the first guidance in the series published in Aug 2021 on “The application of flow cytometry”.

Both guidance documents are available through the British Pharamcopeia.

EUROPE

European Directorate for the Quality of Medicines (EDQM)

Upcoming Event: Quality requirements for nanomedicines 7&8^th June 2022

The EDQM is holding a symposium event to discuss the “Quality requirements for nanomedicines: what role should the European Pharmacopoeia play?”. The COVID-19 pandemic and the emergence of mRNA vaccines have highlighted the importance of nanoparticle formulations – especially lipid-based systems – used for nucleic acid-based APIs. The success of SARS-CoV-2 vaccination programmes around the globe has demonstrated that these nanoparticle-based formulations can be used to produce safe and efficacious medicinal products.

The aim of this event is to identify any gaps and opportunities for standards concerning modern nanoparticle-based formulations which can be filled by the European Pharmacopoeia (Ph. Eur.), notably by setting common quality standards across Europe and beyond.

European Medicines Agency (EMA)

CHMP adopt a positive opinion for CARVYKTI (ciltacabtagene autoleucel)

On 24 March 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product CARVYKTI, intended for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy.

The opinion and supporting documents can be found here.

EMA publishes an Analysis of the first five years of PRIME

PRIME was set up in March 2016 to provide early and enhanced scientific and regulatory support to medicines that have the potential to significantly address patients’ unmet medical needs. This report provides an analysis of the first 5 years of the scheme. From an ATMP perspective, it is exceptionally positive to see that:

• “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)”.
• “Despite their complexity, the 7 advanced therapies (ATMPs) that benefitted from PRIME support and were granted marketing authorisation had on average shorter active assessment time and clock-stop duration than the average assessment time for all types of new active substances in 2020”.

The report also makes a number of recommendations for improvements to the scheme in three areas:

• Timing for entering the PRIME scheme
• Flexibility of scientific advice procedures for PRIME medicines
• Leveraging the knowledge built during development for more robust marketing authorisation applications that can be assessed in an accelerated manner

A toolbox with guidance for developers of medicines supported by EMA’s PRIME scheme on quality data is currently being finalised, following a public consultation that took place last year.

The full report can be found here.

EU National Competent Authority Updates

FRANCE has implemented new GMO procedures since 01 Jan 2022.

Following the publication of Ordinance No. 2021-1325 of October 13, 2021 reforming the assessment of biotechnologies and simplifying the procedure applicable to the confined uses of genetically modified organisms (GMOs) presenting no or negligible risk, new procedures for examining GMO applications have been in place since 01 Jan 2022. In brief, for Class I GMOs, the there is now a centralised procedure for obtaining the relevant site approvals, which can be submitted by the Sponsor (or Sponsor’s delegate) through the DUO platform. From June 2022, the submission procedure is expected to change further for Class I GMOs, with submissions occurring directly to ANSM, and the DUO platform for these submissions being retired (more guidance will be issued closer to the change over date).

More information can be found here.

USA

Food and Drug Administration (FDA)

FDA approves CARVYKTI (ciltacabtagene autoleucel)

On 28^th February 2022, the FDA has approved CARVYKTI (ciltacabtagene autoleucel) for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monclonal antibody.

The related product information and supporting documents can be found here.

FDA releases two new Cell and Gene Therapy related draft Guidances for Industry

The FDA has released two new cell and gene therapy related guidance documents. Industry is encouraged to submit either electronic or written comments on the draft guidance by June 14, 2022 to ensure that the Agency considers any comments prior to commencing work on the final versions.

• Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products; Draft Guidance for Industry
• Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry

Cellular, Tissue, and Gene Therapies Advisory Committee Meeting

The 71^st meeting of the cellular, tissue and gene therapis advisory committee was held on 10 March 2022.

The meeting information, including a watch on-demand recording of the meeting, can be found here.

National Institutes of Health (NIH)

NIH launches program to offer molecular characterization of childhood cancers

The National Cancer Institute, part of the National Institutes of Health, has launched the Molecular Characterization Initiative for pediatric tumors. This program offers tumor molecular characterization, also called biomarker testing, to children, adolescents, and young adults with newly diagnosed central nervous system tumors who are being treated at hospitals that are affiliated with the Children’s Oncology Group (COG)(link is external), an NCI-supported clinical trials group that includes more than 200 hospitals and institutions that treat most children diagnosed with cancer in the United States.

The ambition is that a precise diagnosis, based on the molecular characteristics of a patient’s tumor, will help doctors choose the most effective and potentially least toxic treatment for each child. Data on the molecular changes seen across childhood cancers can also help researchers better understand the molecular causes of childhood cancers and accelerate the development of new, more effective, and potentially less toxic treatments, especially for rare childhood cancers for which treatment options are limited.

NIH’s All of Us Research Program Releases First Genomic Dataset of Nearly 100,000 Whole Genome Sequences

Nearly 100,000 highly diverse whole genome sequences are now available through the National Institutes of Health's All of Us Research Program. About 50% of the data is from individuals who identify with racial or ethnic groups that have historically been underrepresented in research. This data will enable researchers to address yet unanswerable questions about health and disease, leading to new breakthroughs and advancing discoveries to reduce persistent health disparities.

Public Consultations