New Initiatives and Notable Approvals

Last year, the European Medicines Agency (EMA) launched the "Cancer Medicines Pathfinder" initiative to bolster the development and approval of cancer treatments. This initiative focuses on accelerating drug assessments, improving stakeholder dialogue, and effectively communicating the benefits and risks of cancer therapies.

In the first eight months of 2024, the EMA approved 45 new drugs, matching the strong pace set in 2023 when a total of 77 new drugs were approved. Notably, 12 of the drugs approved this year have been granted orphan designation, securing 10 years of market exclusivity with additional incentives for small to medium-sized biopharma firms.

Divergence from FDA Approvals

The divergence between regulatory agencies in the European Union and the United States is notable in the intricate landscape of global pharmaceutical approvals.

For example, Regeneron's Ordspono (odronextamab) was approved by the EMA for blood cancer despite the FDA's rejection five months earlier. The FDA declined to recommend Ordspono due to concerns over the immaturity of its confirmatory trial data. In contrast, the EMA approved Ordspono for two specific types of blood cancers, highlighting a significant difference in regulatory perspectives on the readiness of clinical trial results for market approval.

Similarly, Novo Nordisk's Awiqli (once-weekly insulin icodec), aimed at improving glycemic control in individuals with Type 1 diabetes, faced regulatory hurdles in the United States. An FDA committee voted against recommending Awiqli, citing significant concerns over the increased risk of hypoglycemia compared to the minimal benefits observed in adults with Type 1 diabetes. This decision was primarily based on the outcomes of the ONWARDS 6 trial, where adult patients treated with insulin icodec experienced higher rates of level two and level three hypoglycemia than those receiving once-daily insulin degludec. These findings raised questions about the drug's safety profile and the adequacy of risk mitigation strategies.

Moreover, although Outlook Therapeutics' Lytenava was approved by the EMA, it encountered critical obstacles in the FDA approval process, including several chemistry, manufacturing, and controls (CMC) issues. Observations from pre-approval manufacturing inspections and the demand for further confirmatory clinical evidence led to its rejection by the FDA.

Expansion Beyond New Medicines

In addition to new drug approvals, the EMA also approved 40 expanded indications for existing drugs, such as Dupixent, Eliquis, Tagrisso, and Skyrizi. Furthermore, the agency issued 7 positive opinions in July. For these medicines, formal approvals are expected within a one to two-month timeframe.

Looking Ahead

Oncology often leads in new drug approvals each year. Last year, the EMA issued positive opinions for 25 oncology drugs, representing a third of all new medicines reviewed positively. Stakeholders eagerly anticipate whether this trend will persist, potentially setting new benchmarks in cancer treatment innovation.