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European Biopharmaceutical Review

A New Oncology Era

Classification of cancers is typically based on their histology or tissue of origin, eg, lung cancer, and the development of drugs to treat cancers have followed a histology-specific pathway. Histology-independent drug development represents a new way of defining and treating cancers based on their genetic makeup rather than the tissue of origin. Additionally, the increasing discovery of molecular subtypes of cancer show small subgroups that actually correspond to orphan or ‘niche’ indications, even within larger tumour types.

When a Biomarker Defines the Indication

As the knowledge of tumourigenesis has evolved, oncologists are increasingly categorising organspecific cancers based on molecular markers. One recent announcement in this field was the groundbreaking approval of Keytruda by Merck & Co (pembrolizumab, issued in 2017). This came only a few months after the FDA announced the launch of their new Oncology Centre of Excellence, and it is the first drug approval ever where a biomarker defines the indication. The biomarker indication is approved in the US only so far and is a tissue-agnostic indication in patients with mismatch repair deficiency and microsatellite instability high profiles (1). The scientific rationale underpinning the Keytruda approval has effectively created a single therapeutic approach for patients with different tumour types, allowing extrapolation of the observed treatment effect to diverse tumours. The approval demonstrates a regulatory acceptance of the shift from defining cancers by the site at which they occur towards definition by the molecular changes that drives tumourigenesis. The approval is likely to have implications for how the drug development process is pursued in the future, particularly in oncology, but likely also for other therapeutic areas as science progress for molecularly defined subpopulations thereby enable more targeted and personalised therapies.

Potential Candidates for Tissue-Agnostic Approval

During 2017, several recent events have reflected the FDA’s new regulatory position (apart from pembrolizumab) and the FDA has recently published a Draft Guidance on Targeted Therapies in Low-Frequency Molecular Subsets of a Disease (2-3). Some likely next potential candidates for approval could be larotrectinib and entrectinib, and large collaborative studies (with multiple commercial sponsors) may lead to further approvals:

• Larotrectinib: A tyrosine receptor kinase (Trk) inhibitor (inhibitor of TrkA-, B-, and C-), multiple trials evaluating the drug for efficacy and safety in treating several types of solid tumours are ongoing
• Entrectinib: With orphan drug designations for the treatment of TrkA-, B-, C-, the C-ros oncogene 1, anaplastic lymphoma kinase-positive non-small cell lung cancer and metastatic colorectal cancer and a rare paediatric disease designation for the treatment of neuroblastoma
• The Targeted Agent and Profiling Utilisation study, the first trial ever conducted by the American Society of Clinical Oncology: The study is a non-randomised, later-stage cancer trial (efficacy and safety) that looks at commercially available, targeted anticancer drugs for the treatment of patients with advanced cancer that has a potentially actionable genomic variant. The trial focusses on whether specific targeted therapies can benefit more patients and lead to more personalised therapies
• The National Clinical Trials Molecular Analysis for Therapy Choice: This is a biomarker-driven trial (with 18+ arms and 12+ drugs, both approved and unapproved). In this trial, the patients are assigned treatment based on the genetic changes found in their tumours through genomic sequencing (and other tests)

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Dr Terese Johansson is a Molecular Biologist by original training with a PhD in oncology (rare cancers). After leaving academia, she has been working for MPA, EMA, and AstraZeneca among others. She joined NDA as a regulatory consultant in 2015.
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Dr Terese Johansson
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