In 2024, the FDA granted approval to a variety of groundbreaking treatments, including gene and cell therapies, targeted biologics, and novel small molecules. Among the notable approvals were Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel), the first gene therapy for metachromatic leukodystrophy, and AstraZeneca’s Voydeya (danicopan), a complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria.

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These treatments, along with others, targeted conditions with significant unmet medical needs, such as Alzheimer’s, rare genetic disorders, and advanced cancers. Looking ahead to 2025, the pharmaceutical industry is set to introduce another wave of first-in-class drugs that promise to bring entirely new mechanisms of action. These upcoming approvals are poised to further revolutionize treatment options. Here’s a preview of four groundbreaking drugs that are expected to gain FDA approval in 2025 ...