Institute Report
Expanding Options for Emerging Biopharma in the U.S.: A Decade of Change
Oct 29, 2025

Report Summary:

Emerging biopharma companies – those with R&D spending less than $200Mn and revenues less than $500Mn a year – have become central to biomedical innovation over the past decade, driving the majority of novel drug launches, notably in specialty, orphan, and first-in-class categories. Their agility, resilience, strategic diversity, and focus on unmet needs position them as engines of progress in the health system.

Increasingly these companies are exploring commercial markets on their own and there have been demonstrated successes with these efforts. However, challenges remain in funding, commercialization, and achieving sustained market success.

Key Findings:

  • Emerging biopharma companies – those with R&D spending less than $200Mn and revenues less than $500Mn a year – have become central to biomedical innovation over the past decade, driving the majority of novel drug launches, notably in  specialty, orphan, and first-in-class categories. Their agility, resilience, strategic diversity, and focus on unmet needs position them as engines  of progress in the health system. Increasingly these companies are exploring commercial markets on their  own and there have been demonstrated successes with these efforts. However, challenges remain in funding, commercialization, and achieving sustained market success.
  • Funding for EBPs continues and increasingly includes a focus on China-originated assets. Large ($2Bn+) deals in 2025 through July exceeded the full year 2024 total and are set for another large year with a focus on oncology, obesity, neurology, and AI.
  • EBPs demonstrate higher productivity than larger companies, driven by less complex and faster trials despite lower success rates.
  • EBPs commercialized 197 of 497 U.S. NAS launches in the past decade, including 205 drugs through the abbreviated 505(b)(2) pathway. Their share of launches has grown, reaching 46% of NAS and 56% of 505(b)(2) launches from 2020–2024. U.S.-headquartered EBPs represented 74% of U.S. EBP NAS launches in the past 10 years, and 80% of 505(b)(2)s.
  • Post-launch, EBPs often continue to market drugs, out-license, or are acquired. Of the 197 NAS initially commercialized by 176 EBPs, 69 companies retained the drug, 58 were subsequently acquired, and 21 companies out-licensed the launched drug while remaining in operation for the development and commercialization of other assets. Acquisitions of EBPs following product launch were valued at more than $171Bn, with notable deals including AstraZeneca’s acquisition of Alexion for $39Bn and Gilead’s acquisition of Immunomedics for $21Bn.
  • Launch success across product types is most often associated with expansive plans to demonstrate efficacy to diverse stakeholders. Evidence development beyond initial approval, realistic product assessments, and efficient promotional activity are key. Follow-on approvals for new indications, populations, or formulations drive sustained growth, though not all drugs are suited for such opportunities.
  • A set of common definitions is helpful to fully understand the role that emerging biopharma companies play relative to other participants in the global biopharmaceutical sector. Defining companies by revenue and pipeline activity offers a simple yet effective way of mapping types of companies.
  • For the purposes of this report, segments are determined based on the 2024 calendar year with the exception of analysis of drug origination which assesses companies at the time of origination (first patent filing or first clinical trial).
  • Emerging biopharma (EBP) companies are defined as those with either R&D spend <$200Mn or prescription sales up to $500Mn. Companies with any active pipeline since 2014 were included. Commercial EBP (310)and pre-commercial EBP companies (1,447) are distinguished by the presence of sales or not.
  • The number of novel active substances launched globally has increased significantly over the past 20 years, more than doubling in 2020-2024 compared to 2005–2009.
  • Emerging biopharma companies were the originators of more than half of the 1,005 NAS over the past 20 years, and have been responsible for most of the increase in the overall total.
  • In 2005-2009 EBP companies originated 68 NAS, 43.5% of the NAS in that period. In 2020–2024 EBP originators accounted for 65% (258) of the 394 in the past five years, 58% (189) excluding China-only launches.
  • Over the past decade, EBP companies were responsible for FDA filing and initial launch of 197 novel active substances (NAS) and 205 drugs approved through the abbreviated 505(b)(2) pathway, and annual variability is high for both types of launches.
  • Of the 273 U.S. NAS launches in the past five years, EBP were the FDA filing company for 126. For the 232 launches using the 505(b)(2) abbreviated pathway, EBPs filed 130 of them.
  • Even as the number of NAS and 505(b)(2) launches have been increasing, the share of those launched by EBP companies has been growing even faster.
  • Commercialization by EBPs is followed by important changes for the companies involved, with some continuing to market those medicines, with mixed success, while others sell the company or license the drug to another company, and others ultimately fail.
  • Of the 197 NAS initially commercialized by 176 EBP companies in the past10years, 69 of the companies have retained the drug and continued to operate, while the remainder faced some sort of change.
  • Larger companies acquired 58 of the companies at some point after launch, and 21 of the companies out-licensed the drug(s) to a larger company.
  • Across both novel active substances and 505(b) (2) products from EBP sponsors, key factors are consistently associated with successful launches.
  • As with any launch, product differentiation alone is not the driver of success. Sponsors must plan for and invest in the kinds of evidence, and supportive resources needed by stakeholders both at initial launch and in the periods that follow.
  • Evidence development beyond initial approval is key both for additional approved uses, but also to reflect that competitors are also active with their own products.

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