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How a change in FDA guidance might revolutionise MASH drug development

Transformative new shifts have taken place in the last month in the metabolic dysfunction-associated steatohepatitis (MASH) therapy space, with Madrigal’s Rezdiffra becoming the first approved drug in the EU for patients with MASH and moderate-to-advanced fibrosis (F2–3). Shortly after, Novo Nordisk’s Wegovy was approved in the US for MASH F2–3, providing the first direct competition to Rezdiffra, which until now had exclusive access to US patients since its approval in March 2024. However, these two assets may not be alone in the MASH market for long. A new decision by the FDA has paved the way for even further expansion of the MASH therapy landscape.

Last month, the FDA announced that it would accept Echosens’ letter of intent (LOI) for the qualification of a novel reasonably likely surrogate endpoint (RLSE)—Liver Stiffness Measurement (LSM) by Vibration-Controlled Transient Elastography (VCTE)—in future MASH F2–3 clinical trials. Echosens manufactures a device, the FibroScan, which measures LSM by VCTE.

The impact of this announcement could be transformational for future MASH clinical trials, driving a shift away from the use of liver biopsies in diagnosis and assessment of disease progression. This is particularly notable, given that many past MASH assets had struggled in reaching a biopsy-based fibrosis improvement trial endpoint. Until now, this posed an obstacle to approval, which typically required meeting this endpoint. However, now that LSM by VCTE is on the path to be formally recognised as an RLSE, it is likely that assets could instead focus on achieving improvements in this non-invasive parameter instead. Moreover, LSM by VCTE is far less subjective than pathologist-based biopsy assessment, potentially allowing for greater standardisation of patient outcomes.

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As well as this, the ability to avoid biopsy altogether will likely drastically speed up trial recruitment times, given the difficulty of conducting biopsies as opposed to measuring LSM by VCTE. As Echosens noted in its press release, this could “accelerate drug development and also be easily translatable to clinical practice.”

It is important to note that the LOI is only the first step in the FDA’s complicated Drug Development Tool (DDT) qualification process. Next, Echosens must make a Qualification Plan (QP) submission with available relevant data, knowledge gaps, proposed data collection plans, and analysis plans, which the FDA will take up to six months to review. After that, the FDA will issue a Determination Letter that will make additional requests for data and include recommendations about data required for the third step, the Full Qualification Package (FQP). The FQP will include detailed descriptions of all studies, analyses, and results related to LSM by VCTE, which will require a comprehensive, up to ten-month review by the FDA. After this, the FDA issues another Determination Letter that decides whether it has qualified. Nevertheless, the fact that the FDA chose to issue a press release about the acceptance of the first step alone indicates a high level of confidence in LSM by VCTE’s future success in this process.


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