Cancer Holds Pharma R&D Lead as Obesity Pipeline Swells 30.7% and Rare Disease Share Rises
核心洞察
Oncology still accounts for 38.6% of newly identified drug candidates, but the number of cancer drugs in development fell 4.6% for a second consecutive year.
The anti-obesity (搜索) category grew 30.7% to 588 drugs, and obesity entered the top 10 indications for the first time with 576 candidates.
Rare disease drugs slipped 1.3% to 7,618 candidates yet rose to 33.2% of the overall pipeline, with Novartis leading at 116 candidates.
Oncology remains the dominant force in pharmaceutical research and development, but its grip is loosening as obesity (搜索) and rare disease programs expand, according to the Pharma R&D Annual Review 2026, a Norstella report drawn from Citeline clinical data.
Cancer still accounts for the largest number of pipeline candidates, yet the number of oncology drugs in development has fallen for the second year in a row after almost a decade of solid growth, declining 4.6% year over year. Oncology represented 38.6% of newly identified drug candidates in 2025, and remains the target of nearly two out of five drugs in development.
Overall, there are fewer drugs in the pipeline this year, marking the first drop since the mid-1990s. 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.
Lung Cancer Overtakes Breast Cancer in the Pipeline
Cancers represented seven of the top 10 diseases or indications in development and the entire top six. Non-small cell lung cancer (搜索) took the top spot overall, overtaking breast cancer for the first time, with colorectal, pancreatic, ovarian and prostate cancers rounding out the top six.
Lung cancer R&D is active across subtypes. Amgen's first-in-class bispecific small cell lung cancer (搜索) drug, Imdelltra, won accelerated FDA approval in 2024 and full approval in late 2025. Amgen also said a phase 3 trial of Imdelltra in combination with Imfinzi showed "landmark" improvement in overall survival in first-line extensive-stage small cell lung cancer, though the company has not yet disclosed detailed results.
BioNTech has 16 ongoing lung cancer trials, including five pivotal phase 3 and two novel-novel combination trials. One of its pipeline stars is pumitamig (搜索), which the company is developing with Bristol Myers Squibb (搜索). BioNTech is presenting data about several lung cancer trials this month, including one combining pumitamig with elfetabart drozuntecan (搜索), which BioNTech said will provide the first lung cancer data for a PD-(L)1xVEGF (搜索) bispecific immunomodulator combined with an antibody-drug conjugate.
Obesity Breaks Into the Top 10
Fueled by blockbuster drugs and new oral formulations, the anti-obesity (搜索) 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. The GLP-1 receptor (搜索) was the No. 2 drug target in development, behind the cancer target epidermal growth factor receptor (搜索).
The two biggest obesity (搜索) players, Eli Lilly and Novo Nordisk, are both trying to outdo themselves with next-generation 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 (搜索), 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 Disease Share Grows Despite Mixed Readouts
Rare disease drug developers have notched significant regulatory wins in the past year, from global regulators allowing AMO Pharma (搜索) to use hospitalization as the primary efficacy endpoint in its registration trial for a rare neuromuscular disorder to the FDA approving Stealth BioTherapeutics (搜索)' Forzinity (搜索) despite its tiny trial size.
That 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%. The share of newly identified candidates targeting rare diseases slipped from 20% to 19.4%.
Among the top 20 companies, Novartis has the most rare disease drugs in development with 116, while Amgen has the highest percentage of rare disease candidates, with rare diseases making up 52.2% of its pipeline.
Novartis' rare disease pipeline suffered a major setback this week when its phase 3 del-desiran trial in myotonic dystrophy type 1 (搜索) missed its primary endpoint measuring video hand-opening time. Amgen scrapped one Sjögren's syndrome (搜索) drug, adezkibart (搜索), after an interim analysis found the phase 2 trial was unlikely to meet its primary endpoint, but it is continuing to develop its phase 3 candidate, dazodalibep, for the condition.