Company lists · Data refreshed September 2026

Top 25 Rare disease pharmaceutical companies based in the United States

Name: Rare disease pharmaceutical companies based in the United States
Creator: RevenueBase
License: https://creativecommons.org/licenses/by/4.0/

Compiled by the RevenueBase Data Team Data refreshed Sep 17, 2026 362 companies tracked

RevenueBase identified 362 rare disease pharmaceutical companies headquartered in the United States as of September 2026 and tracks 190,245 professional contacts across them, including 81,950 with verified work emails. New York is the largest hub.

This list covers rare disease pharmaceutical companies headquartered in the United States, matched on what each company actually does as described in its own words. Ranked by team size, largest first. Every row shows the company's own description, its RevenueBase Smart Search rating, and when it was last checked. Data refreshed September 17, 2026.

Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase Smart Search, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.

How to read this list HQ checked on every row RevenueBase Smart Search rating on every row Refreshed Sep 17, 2026 86% judged on-target in blind review

362Companies

190,245Contacts tracked

81,950Verified emails

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The 25 largest rare disease pharmaceutical companies based in the United States

Ranked by team size

Top 25 of 362 rare disease pharmaceutical companies based in the United States, ranked by team size, largest first. Data refreshed Sep 17, 2026. RevenueBase Smart Search rates how each company's information relates to rare disease pharmaceutical companies: strong evidence (its own description names it), partial (names part of it), indirect (matched on the rest of its profile rather than the description shown). Click a contact count to view that company's people.
#	Company	RevenueBase Smart Search	Headquarters	Employees	Contacts
1	Bristol Myers Squibb	Indirect evidence	Lawrence Township, NJ, United StatesHQ checked Sep 17, 2026	10001+	36,379 contacts →
	Indirect evidence “At Bristol Myers Squibb, we work every day to transform patients’ lives through science.” Source: RevenueBase Database · checked Sep 17, 2026
2	Johnson & Johnson Innovative Medicine	Indirect evidence	Titusville, NJ, United StatesHQ checked Sep 17, 2026	10001+	26,637 contacts →
	Indirect evidence “At Johnson & Johnson Innovative Medicine, we innovate with purpose, to lead where medicine is going.” Source: RevenueBase Database · checked Sep 17, 2026
3	Genentech	Indirect evidence	South San Francisco, CA, United StatesHQ checked Sep 17, 2026	10001+	17,217 contacts →
	Indirect evidence “About Genentech We're passionate about finding solutions for people facing the world's most difficult-to-treat conditions.” Source: RevenueBase Database · checked Sep 17, 2026
4	Regeneron	Indirect evidence	Tarrytown, NY, United StatesHQ checked Sep 17, 2026	10001+	16,043 contacts →
	Indirect evidence “At Regeneron we believe that when the right idea finds the right team, powerful change is possible.” Source: RevenueBase Database · checked Sep 17, 2026
5	Biogen	Indirect evidence	Cambridge, MA, United StatesHQ checked Sep 17, 2026	5,001-10,000	10,389 contacts →
	Indirect evidence “Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for…” Source: RevenueBase Database · checked Sep 17, 2026
6	Vertex Pharmaceuticals	Partial evidence	Boston, MA, United StatesHQ checked Sep 17, 2026	5,001-10,000	8,099 contacts →
	Partial evidence “Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and has made significant…” Source: RevenueBase Database · checked Sep 17, 2026
7	Alexion Pharmaceuticals, INC	Strong evidence	Boston, MA, United StatesHQ checked Sep 17, 2026	1,001-5,000	6,136 contacts →
	Strong evidence “Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Source: RevenueBase Database · checked Sep 17, 2026
8	Biomarin Pharmaceutical INC	Indirect evidence	San Rafael, CA, United StatesHQ checked Sep 17, 2026	1,001-5,000	3,943 contacts →
	Indirect evidence “Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Source: RevenueBase Database · checked Sep 17, 2026
9	Eisai US	Indirect evidence	Nutley, NJ, United StatesHQ checked Sep 17, 2026	1,001-5,000	2,570 contacts →
	Indirect evidence “At Eisai Inc, human health care (hhc) is our goal.” Source: RevenueBase Database · checked Sep 17, 2026
10	EMD Serono, INC	Indirect evidence	Boston, MA, United StatesHQ checked Sep 17, 2026	1,001-5,000	1,578 contacts →
	Indirect evidence “We aspire to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer.” Source: RevenueBase Database · checked Sep 17, 2026
11	Syneos Health Clinical Solutions	Indirect evidence	Morrisville, NC, United StatesHQ checked Sep 17, 2026	10001+	1,569 contacts →
	Indirect evidence “Syneos Health® is a leading fully integrated biopharmaceutical solutions organization built to accelerate customer success.” Source: RevenueBase Database · checked Sep 17, 2026
12	Ultragenyx	Indirect evidence	Novato, CA, United StatesHQ checked Sep 17, 2026	1,001-5,000	1,556 contacts →
	Indirect evidence “Leading with purpose.” Source: RevenueBase Database · checked Sep 17, 2026
13	Insmed Incorporated	Partial evidence	Bridgewater Township, NJ, United StatesHQ checked Sep 17, 2026	1,001-5,000	1,512 contacts →
	Partial evidence “Insmed is a people-rst global biopharmaceutical company striving to deliver rst- and best-in-class therapies to transform the lives of patients facing serious diseases.” Source: RevenueBase Database · checked Sep 17, 2026
14	PTC Therapeutics, INC	Strong evidence	Warren, NJ, United StatesHQ checked Sep 17, 2026	501-1,000	1,212 contacts →
	Strong evidence “PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Source: RevenueBase Database · checked Sep 17, 2026
15	Purdue Pharma L.P	Indirect evidence	Stamford, CT, United StatesHQ checked Sep 17, 2026	501-1,000	885 contacts →
	Indirect evidence “Purdue and its subsidiaries develop, manufacture and market medications to meet the evolving needs of healthcare professionals, patients, and caregivers.” Source: RevenueBase Database · checked Sep 17, 2026
16	Bridgebio	Indirect evidence	Palo Alto, CA, United StatesHQ checked Sep 17, 2026	501-1,000	837 contacts →
	Indirect evidence “We exist to bring transformative medicines to people living with genetic conditions.” Source: RevenueBase Database · checked Sep 17, 2026
17	Apellis Pharmaceuticals	Indirect evidence	Waltham, MA, United StatesHQ checked Sep 17, 2026	501-1,000	823 contacts →
	Indirect evidence “Apellis is now part of Biogen.” Source: RevenueBase Database · checked Sep 17, 2026
18	Azurity Pharmaceuticals	Partial evidence	Woburn, MA, United StatesHQ checked Sep 17, 2026	501-1,000	790 contacts →
	Partial evidence “Azurity Pharmaceuticals is a privately held global pharmaceutical company dedicated to redefining medicine for the real world.” Source: RevenueBase Database · checked Sep 17, 2026
19	Kyowa Kirin, Inc.- U.S	Partial evidence	Princeton, NJ, United StatesHQ checked Sep 17, 2026	501-1,000	727 contacts →
	Partial evidence “Kyowa Kirin is a global specialty pharmaceutical company that applies state-of-the-art biotechnologies to discover and deliver novel medicines.” Source: RevenueBase Database · checked Sep 17, 2026
20	Arrowhead Pharmaceuticals	Strong evidence	Pasadena, CA, United StatesHQ checked Sep 17, 2026	501-1,000	657 contacts →
	Strong evidence “Arrowhead Pharmaceuticals is a clinical stage, Nasdaq listed (ticker ARWR) company developing medicines that treat intractable diseases by silencing the genes that cause them.” Source: RevenueBase Database · checked Sep 17, 2026
21	ANI Pharmaceuticals, INC	Partial evidence	Princeton, NJ, United StatesHQ checked Sep 17, 2026	501-1,000	625 contacts →
	Partial evidence “ANI Pharmaceuticals, Inc.(Nasdaq: ANIP) is a diversified biopharmaceutical company committed to its mission of “Serving Patients, Improving Lives" by developing, manufacturing…” Source: RevenueBase Database · checked Sep 17, 2026
22	Agios Pharmaceuticals	Strong evidence	Cambridge, MA, United StatesHQ checked Sep 17, 2026	501-1,000	603 contacts →
	Strong evidence “Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Source: RevenueBase Database · checked Sep 17, 2026
23	Pantherx Rare Pharmacy	Strong evidence	Pittsburgh, PA, United StatesHQ checked Sep 17, 2026	501-1,000	582 contacts →
	Strong evidence “PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Source: RevenueBase Database · checked Sep 17, 2026
24	Vanda Pharmaceuticals	Indirect evidence	Washington, DC, United StatesHQ checked Sep 17, 2026	201-500	575 contacts →
	Indirect evidence “Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the…” Source: RevenueBase Database · checked Sep 17, 2026
25	Amag Pharmaceuticals	Indirect evidence	Waltham, MA, United StatesHQ checked Sep 17, 2026	201-500	548 contacts →
	Indirect evidence “At AMAG, aiming higher is in our DNA.” Source: RevenueBase Database · checked Sep 17, 2026

RevenueBase Smart Search

RevenueBase Smart Search assesses information from a company’s LinkedIn profile, information supplied directly by the company, its website, and other publicly available sources. It uses that information to rate how closely the company relates to this list’s category: rare disease pharmaceutical companies. Every row carries its rating and the description the rating was read from, so each one can be checked rather than taken on trust.

Strong evidence: The company's own public description of its business names rare disease pharmaceutical companies.
Partial evidence: Its own public description names part of rare disease pharmaceutical companies, so it works in or next to the category without describing itself that way.
Indirect evidence: The description shown does not name rare disease pharmaceutical companies. The match rests on the rest of the company's public profile rather than the one sentence quoted here, so this is the rating to check first.

RevenueBase Smart Search is how these lists are built: RevenueBase's data engine and AI assemble the companies that match a category defined in a user's own words, so a list can be about what companies actually do rather than about whichever industry code they were filed under.

Showing 25 of 362 rare disease pharmaceutical companies based in the United States

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Rare disease pharmaceutical companies based in the United States at a glance

As of Sep 17, 2026

43.1%Verified-email coverage

525.5Average contacts per company

11-50Median size (employees)

5.0%Largest-city concentration · New York

Sep 17, 2026Page data refreshed

Companies by employee count

1-10

124

11-50

107

51-200

201-500

501-1,000

1,001-5,000

5,001-10,000

10001+

Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 362 companies.

How this list was built

Company categories are rarely black and white. RevenueBase reads each company's own description of its business to decide whether it belongs among rare disease pharmaceutical companies, so some rows will be adjacent to the category rather than pure plays. The description and RevenueBase Smart Search rating on every row are there so you can check each match yourself.

Source: The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase Smart Search, the natural-language company search available to every RevenueBase user.
What qualifies: Companies headquartered in the United States whose own description of what they do matches “rare disease pharmaceutical companies”. Matching reads each company's description, not its industry code or its name.
RevenueBase Smart Search: RevenueBase Smart Search is how this list is assembled and how each row is rated. RevenueBase's data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to a category defined in a user's own words. Each row is rated strong evidence (its own description names rare disease pharmaceutical companies), partial evidence (it names part of the category) or indirect evidence (the description shown names none of it and the match rests on the rest of the company's profile). The description supporting each rating is quoted on the row.
What we exclude: Companies whose own description does not clearly place them among rare disease pharmaceutical companies, and records with no verified work email. Companies headquartered outside United States are excluded even when they keep an office there.
Match quality: In blind human judging of this matching method, 86% of matched companies were on target. Expect roughly 1 in 7 rows to be adjacent to the category rather than a pure play. Every row shows the company's own description and its RevenueBase Smart Search rating so you can judge each match yourself. Read the full methodology: how the lists are built, how the judging worked, and what the figure means.
Ranking: Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
Verification: Every listed company carries at least one verified work email, 81,950 across the full list. Contacts re-verify on a rolling cadence, and each contact's own verification date is shown inside RevenueBase.
Refresh: This page's figures were pulled from the company graph on September 17, 2026. Lists refresh periodically as the graph is re-checked.
Corrections: Wrong category, wrong headquarters, or a company that has closed? Use “Report an issue” on any row. Reports go to the RevenueBase Data Team and feed back into the company graph.

Frequently asked

What is RevenueBase Smart Search?

RevenueBase Smart Search is how RevenueBase decides which companies belong on this list and how strongly each one fits. Its data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to the category the list is about. Each row is rated strong evidence when the company's own description names rare disease pharmaceutical companies, partial evidence when it names part of the category, and indirect evidence when the description shown names none of it and the match rests on the rest of the company's profile.

How many rare disease pharmaceutical companies are there based in the United States?

RevenueBase identified 362 rare disease pharmaceutical companies headquartered in the United States as of September 2026, matched on each company's own description of what it does rather than its industry code.

What is the largest company on this list?

Bristol Myers Squibb, headquartered in Lawrence Township, tops this list with the largest team on record.

Which city has the most rare disease pharmaceutical companies?

New York leads with 18 headquartered companies, followed by Boston (17) and San Diego (13).

How do I get contact information for these companies?

A free RevenueBase account lets you run this exact search and work the top results, verified emails and direct dials included. Upgrading unlocks all 81,950 verified decision-maker contacts tracked across these 362 companies.

How current is this list?

The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 17, 2026.

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About this data

RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase Smart Search, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.