The 2023 oncology medicine approvals from the European Medicines Agency (EMA)

by | May 14, 2024 | European Medicines Agency, Executive Summary, Marketing Authorisation Application

Highlights

  • Overview of conditional marketing authorisations (CMAs) granted for oncology medicines in 2023.
  • Orphan maintenance requirements for oncology medicines in 2023.
  • Paediatric requirements for oncology medicines in 2023.

Introduction

During 2023, the European Medicines Agency (EMA) recommended 77 medicines for marketing authorisation – 39 of which had a new active substance not previously authorised in the European Union (EU). The most common therapeutic area was for the treatment of cancer, including new medicines for both solid and haematological tumours.

This executive summary focuses specifically on the oncology medicine approvals and in particular, those that were approved under a CMA.

Conditional marketing approvals

In 2023, a recommendation for a CMA was granted by the Committee for Medicinal Products for Human Use (CHMP) for:

  • Columvi (glofitamab),
  • Elrexfio (elranatamab),
  • Jaypirca (pirtobrutinib),
  • Krazati (adagrasib)
  • Lytgobi (futibatinib),
  • Talvey (talquetamab), and
  • Tepkinly (epcoritamab).

These approvals were all granted from single-arm trials and all of these CMAs followed similar development themes.

  1. Patients had limited treatment options – either having broadly defined relapsed or refractory disease, or having received a specified number of prior therapies.
  2. The pivotal efficacy data was derived from a single Phase 1/2 study (single-arm).
  3. The primary endpoint in each case was response rate (RR) – which was deemed to be a clinically meaningful measure of efficacy in these patients with relapsed or refractory cancer (albeit the definition varied by indication).

We have created a whitepaper with detailed information on the oncology products with CMAs in 2023, including pivotal trial data and post-approval commitments. Download it to read more.

Orphan designation challenges

Many of the medicines were recommended for approval in conditions which satisfy orphan criteria in the EU. Orphan criteria are principally based on rarity (prevalence of fewer than five patients in 10,000 population) and the condition that the proposed treatment is for a life-threatening or debilitating condition.

At the time of filing a Marketing Authorisation Application (MAA), the sponsor is required to submit an orphan maintenance report, which demonstrates that orphan criteria are still met to maintain an orphan designation.

Due to the number of therapies available in certain oncological and hematological conditions, the ability to demonstrate significant benefit at the time of filing a Marketing Authorisation Application (MAA) has become quite complex.

Paediatric requirements

In the EU, a precursor to submitting an MAA is that according to the Paediatric Regulation, the sponsor must agree a paediatric investigation plan (PIP) with the Paediatric Committee (PDCO), which details the clinical trials that must be completed for use of the product in the paediatric population.

Full (product or class) or partial waivers are possible if the condition does not occur in children or if the medicinal product will likely be ineffective or unsafe in a paediatric population. It is also possible to defer the completion of measures until after you submit your MAA.

Some of the 2023 oncology CMAs were granted product-specific waivers.

How Somerville Development Partners can help

We specialise in oncology and hematology.

We support a complete spectrum of regulatory activities for new cancer medicines that target rare, biomarker-driven subsets, and utilise Phase 1/2 or Phase 2 studies that become the pivotal efficacy and safety registrational clinical trials.

Get all the details of oncology approvals from the EMA in 2023, as well as detailed information on the specific CMAs granted and what it takes to maintain orphan designation in cancer therapies.

Read the Full Oncology Approvals Whitepaper

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Nicole Brooks, Regulatory Consultant / Copywriter
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