Cancer still dominates pharma pipelines by a wide margin, but its share slipped for the second year in a row with its pipeline numbers shrinking by 4.6% from the year before. But cancer is far from alone. There are fewer drugs in the pipeline altogether this year, marking the first drop since the mid-1990s, according to the Pharma R&D Annual Review 2026, a Norstella report drawn from Citeline clinical data.
Citeline cautioned, however, that changes to its data system likely exaggerated the year-over-year decline and said the overall pipeline has probably been fairly flat in recent years.
Still, some therapeutic areas and disease state pipelines are bucking the downward trend, including obesity, which cracked the top 10 disease states for the first time. And while rare diseases’ share of newly identified candidates slipped from 20% to 19.4%, the share of all pipeline drugs targeting at least one rare disease rose from 32.3% to 33.2%.
Cancer still reigns supreme
Although cancer still accounts for the largest number of pipeline candidates, the number of oncology drugs in development has fallen for the second year in a row after almost a decade of solid growth.
Still, cancer likely won’t lose its overall dominance, with oncology the target of nearly two out of five drugs in development. Cancers represented seven of the top 10 diseases or indications in development and the entire top six, with non-small cell lung cancer taking the top spot overall and overtaking breast cancer for the first time. Colorectal, pancreatic, ovarian and prostate cancers rounded out the top six.
Lung cancer R&D is active across subtypes. Amgen’s first-in-class bispecific small cell lung cancer drug, Imdelltra, won accelerated FDA approval in 2024 and full approval in late 2025. BioNTech, meanwhile, has 16 ongoing lung cancer trials, including five pivotal phase 3 and two novel-novel combination trials.
One of its pipeline stars is pumitamig, which the company is developing with Bristol Myers Squibb. BioNTech is presenting data about several lung cancer trials this month, including one combining pumitamig with elfetabart drozuntecan, which BioNTech said will provide the first lung cancer data for a PD-(L)1xVEGF bispecific immunomodulator combined with an antibody-drug conjugate.
Elsewhere, Amgen said a phase 3 trial of Imdelltra in combination with Imfinzi showed “landmark” improvement in overall survival, according to the company, in first-line extensive-stage small cell lung cancer. Amgen has not yet disclosed detailed results.
Obesity on the rise
Fueled by blockbuster drugs and new oral formulations, the anti-obesity therapeutic category swelled 30.7% this year, to 588 drugs from 450.
Obesity also entered the top 10 specific diseases or indications for the first time, rising from No. 19 last year, with 576 drugs in development compared with 430.
Obesity showed up in other top spots, too. The GLP-1 receptor was also the No. 2 drug target in development, behind the cancer target epidermal growth factor receptor.
The two biggest obesity players, Eli Lilly and Novo Nordisk, are both trying to outdo themselves with their next-gen obesity candidates. Lilly scored in May when the highest dose of its potential blockbuster-in-the-making retatrutide delivered average weight loss of 28.3% at 80 weeks in a phase 3 trial of people without diabetes. Novo Nordisk’s CagriSema, meanwhile, failed to demonstrate noninferiority to Lilly’s tirzepatide in a head-to-head phase 3 trial a few months earlier.
Additionally, Structure Therapeutics is making waves with its own oral GLP-1 candidate, aleniglipron, which produced mean weight loss of up to 16.2% at 72 weeks in a phase 2b open-label extension, with no evidence of a weight loss plateau, according to the company.
Rare diseases grab a big piece of the pie
Rare disease drug developers have notched significant regulatory wins in the past year, from global regulators allowing AMO Pharma to use hospitalization as the primary efficacy endpoint in its registration trial for a rare neuromuscular disorder to the FDA approving Stealth BioTherapeutics’ Forzinity despite its tiny trial size.
That rare disease regulatory focus is reflected in the rare disease pipeline, which stayed relatively steady from last year. Rather than constituting a single therapeutic area, rare diseases span several categories in the report. The number of drugs targeting at least one rare disease fell 1.3% to 7,618, but their share of the overall pipeline rose from 32.3% to 33.2%.
Among the top 20 companies, Novartis has the most rare disease drugs in development with 116. But Amgen has the highest percentage of rare disease candidates with rare diseases making up 52.2% of its pipeline.
However, Novartis’ rare disease pipeline suffered a major setback this week when its phase 3 del-desiran trial in myotonic dystrophy type 1 missed its primary endpoint measuring video hand-opening time. And while Amgen scrapped one Sjögren’s syndrome drug, adezkibart, after an interim analysis found the phase 2 trial was unlikely to meet its primary endpoint, it’s continuing to develop its phase 3 candidate, dazodalibep, for the condition.
