The team behind Biomedtracker at Citeline have scoured the late-stage pipeline for drugs producing pivotal clinical trial data and likely to undergo FDA review and/or launch during 2025. This reveals 69 noteworthy approvals across 66 discrete drugs, including 35 that are deemed to be practice changing.
The final totals will come in higher still, as this exercise focuses on the largest therapeutic advances. As the charts and accompanying infographic show, 2025 is set to be a diverse year that features innovative new therapies with a range of mechanisms across the spectrum of disease areas.
New Launches Will Span The Breadth Of Human Disease
Oncology is the most represented therapy area, continuing the long-standing trend of rapidly improving standards of care. Several new therapies are expected to make their debut, including the first-in-class bispecific antibody against PD-1 and VEGF, ivonescimab.
Summit Therapeutics’s drug is expected to compete initially in non-small cell lung cancer (NSCLC), before expanding into breast cancer. Here it will be joined by several other new launches, including Dato-DXd and inavolisib, alongside a label expansion for an existing standard of care, Enhertu.
In total, 14 different tumor types are likely to see important new treatment options during 2025.
Developments in immunology are centered around expanding product labels. J&J will be aiming to secure approvals for Tremfya in Crohn’s disease and ulcerative colitis with positive data from its GALAXI and QUASAR clinical programs. Similarly, Amgen has expansions in mind for Uplizna myasthenia gravis and immunoglobulin G4-related disease after successful clinical trial results in 2024.
Elsewhere, the potential approval of CagriSema for obesity is a standout. Its REDEFINE 1 clinical trial is one of the most watched studies as attention focuses on whether this drug combination can achieve weight loss in excess of 20% accompanied by a balanced risk-benefit profile. Positive results in late 2024 would set the scene for a potential late 2025 launch.
From obesity – one of the most prevalent diseases – to the other end of the spectrum, several rare diseases may welcome much-needed new treatment options. Elamipretide is under review with a January FDA action date, making it one of the first drugs to look out for.
Its developer Stealth Biotherapeutics has shown encouraging functional data in Barth syndrome, which has no known approved drug therapies. Neurocrine is targeting congenital adrenal hyperplasia with crinecerfont, which would become the first new treatment in 70 years.
Finally, Upstaza is designed to correct the mutation at the heart of AADC deficiency. The rare disease community will hope that PTC Therapeutics will launch this gene therapy in the US in 2025, building upon European launches from 2022.
Molecular Diversity With New Classes Well Represented
Upstaza is not the only innovative gene therapy with a potential US launch in 2025, but is joined by others targeting diseases such as Fanconi anemia and head and neck cancer, alongside an expected label expansion for Zolgensma in spinal muscular atrophy.
Similarly, there are multiple RNA-based drugs slated to launch in 2025, including new entrants into the familial chylomicronemia syndrome market from Ionis and Arrowhead. Sanofi’s fitusiran for hemophilia and Moderna’s mRNA flu vaccine are the others to watch. Both represent potential first-in-class approvals in highly competitive treatment categories, with clinical differentiation that supports blockbuster revenue forecasts.
2025 may be a quieter year on the cell therapy side, although not completely unproductive. Capricor Therapeutics is pursuing a biologics license application for deramiocel as a potential treatment for cardiomyopathy in Duchenne muscular dystrophy.
Around half of the key launches will be for small molecules, demonstrating the enduring appeal of oral pills, and the considerable unmet need that such drugs continue to address. Within the antibody group, there are some notable first-in-class candidates. This includes Astellas’s Vyloy, targeting Claudin 18.2 expressed in gastric cancer, while apitegromab is aiming to become the first monoclonal for spinal muscular atrophy.
The renaissance of the antibody-drug conjugate class will also continue throughout 2025 with a plethora of launches (Dato-DXd, patritumab deruxtecan), relaunches (Blenrep), and label expansions (Enhertu).
Clinical Success Is Only Half The Battle
These examples show that the industry’s efforts in R&D are set to result in hard-won product launches. They are the product of many years of development and investment, and often stand on the shoulders of clinical failures that have preceded them.
Getting to this stage, on the precipice of regulatory approval, is a feat in itself. In order to become truly practice-changing therapies, their developers must also plan for commercialization and patient access. So let’s acknowledge the achievements to date and recognize that new challenges await.
This article is derived from an annual Citeline report for Biomedtracker subscribers titled “Key Potential Drug Launches in 2025”. The report harnesses Biomedtracker’s comprehensive database of drug events and catalysts alongside expert, contextual analyst commentary. Clients can access the report via this link, while potential readers can contact sara.lafever@norstella.com if they would like to learn more.
