Week of Sept. 18, 2023

This week we explore the Food and Drug Administration’s new guidance related to single trial approvals and the National Institutes of Health’s commencement of a trial for a universal flu vaccine. We also look at the start of clinical trials for CRISPR-based leukemia treatment and the FDA’s rejection of a nasal epinephrine spray. In addition, we highlight MedTech Europe’s open letter to address reforms to European medtech regulations.

Each week we highlight five things affecting the life sciences industry. Here’s the latest.

The FDA has issued draft guidance emphasizing the importance of thorough data submission and early communication for biopharmaceutical companies seeking FDA approval based on just one clinical trial, according to Fierce Pharma. While the FDA typically requires two studies for efficacy and safety, exceptions are occasionally made for situations where conducting multiple trials may not be practical. This new guidance underscores the significance of the quality and quantity of clinical trial data. It also encourages companies to engage in early communication with the FDA to justify a single-trial approach and provides alternative options for obtaining confirmatory evidence.

The National Institutes of Health announced this week that it has initiated a Phase 1 clinical trial for a new universal influenza vaccine candidate called FluMos-v2. The trial will assess the vaccine’s safety and its ability to stimulate an immune response. Unlike seasonal influenza vaccines, which target specific strains, this universal vaccine aims to provide protection against a wide range of influenza strains. The trial will enroll 24 healthy volunteers. Its success could contribute to the development of more broadly effective flu vaccines.

A research team has initiated a clinical trial using a CRISPR-based tool to engage in base editing to modify four genes in immune cells for improved tumor targeting in leukemia patients, according to Nature. Base editing offers greater control over DNA changes than traditional CRISPR solutions, although safety concerns persist. Several research groups are studying base-editing technology, exploring its potential in cancer therapy and other applications.

According to NBC News, the FDA announced this week that it will not approve a nasal epinephrine spray.  Epinephrine has been used for over a century to treat anaphylaxis, the most severe type of allergic reaction. This is the first time a needle-free epinephrine delivery system has been reviewed by the FDA.  The system had previously garnered support from the FDA’s advisory committee. It is rare that the final approval decision differs from the opinion of the advisory committee.

MedTech Europe, in collaboration with 34 national associations, has penned an open letter to the European commissioner for Health and Food Safety highlighting the urgent need for a comprehensive overhaul of the European regulatory framework for medical technologies. According to Medical Plastics News, MedTech Europe acknowledges the efforts of the European Commission and the Medical Device Coordination Group in addressing short-term challenges. However, they emphasize that achieving the full objectives of in vitro diagnostics regulation and medical devices regulation necessitates sweeping changes in efficiency, innovation and governance, without compromising patient safety. The organization’s key demands include a more efficient CE marking system (showing the goods’ conformity with European health, safety and environmental protection standards), support for innovation through dedicated assessment pathways, and the establishment of a singular governance structure with the power to make overarching decisions.

For more insights in life sciences, check out RSM’s industry outlook.

Source link: https://realeconomy.rsmus.com/5-things-to-know-in-life-sciences-week-of-sept-18-2023/ by Justin Culbertson at realeconomy.rsmus.com