Company lists · Data refreshed September 2026

Top 25 Orphan drug manufacturers worldwide

RevenueBase identified 625 orphan drug manufacturers worldwide as of September 2026 and tracks 213,365 professional contacts across them, including 85,537 with verified work emails and 0 with mobile numbers. The United States is the largest market and London the largest hub.

This list covers orphan drug manufacturers worldwide, 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. Country and city lists for orphan drug manufacturers follow the table.

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
625Companies
213,365Contacts tracked
85,537Verified emails
0Mobile numbers
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The 25 largest orphan drug manufacturers worldwide

Ranked by team size
Top 25 of 625 orphan drug manufacturers worldwide, ranked by team size, largest first. Data refreshed Sep 17, 2026. RevenueBase Smart Search rates how each company's information relates to orphan drug manufacturers: 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.
#CompanyRevenueBase Smart SearchHeadquartersEmployeesContacts
1Indirect evidenceLawrence Township, NJ, United StatesHQ checked Sep 17, 202610001+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
2Indirect evidenceTokyo, IL, JapanHQ checked Sep 17, 202610001+29,414 contacts →

Indirect evidence

“We all want a healthier future.”

Source: RevenueBase Database · checked Sep 17, 2026
3Indirect evidenceTitusville, NJ, United StatesHQ checked Sep 17, 202610001+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
4Indirect evidenceSouth San Francisco, CA, United StatesHQ checked Sep 17, 202610001+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
5Indirect evidenceTarrytown, NY, United StatesHQ checked Sep 17, 202610001+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
6Indirect evidenceSuresnes, FranceHQ checked Sep 17, 202610001+10,958 contacts →

Indirect evidence

“Servier is an independent international pharmaceutical group governed by a foundation.”

Source: RevenueBase Database · checked Sep 17, 2026
7Indirect evidenceCambridge, MA, United StatesHQ checked Sep 17, 20265,001-10,00010,389 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
8Indirect evidenceBoston, MA, United StatesHQ checked Sep 17, 20261,001-5,0006,136 contacts →

Indirect evidence

“Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.”

Source: RevenueBase Database · checked Sep 17, 2026
9Indirect evidenceParis, FranceHQ checked Sep 17, 20265,001-10,0005,943 contacts →

Indirect evidence

“We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.”

Source: RevenueBase Database · checked Sep 17, 2026
10Indirect evidenceSan Rafael, CA, United StatesHQ checked Sep 17, 20261,001-5,0003,943 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
11Indirect evidenceNutley, NJ, United StatesHQ checked Sep 17, 20261,001-5,0002,570 contacts →

Indirect evidence

“At Eisai Inc, human health care (hhc) is our goal.”

Source: RevenueBase Database · checked Sep 17, 2026
12Indirect evidenceHyderabad, IndiaHQ checked Sep 17, 20261,001-5,0001,601 contacts →

Indirect evidence

“Hetero Healthcare Ltd is a leading Innovative Pharmaceutical Company in India, headquartered at Hyderabad.”

Source: RevenueBase Database · checked Sep 17, 2026
13Indirect evidenceBoston, MA, United StatesHQ checked Sep 17, 20261,001-5,0001,578 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
14Indirect evidenceMorrisville, NC, United StatesHQ checked Sep 17, 202610001+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
15Indirect evidenceNovato, CA, United StatesHQ checked Sep 17, 20261,001-5,0001,556 contacts →

Indirect evidence

“Leading with purpose.”

Source: RevenueBase Database · checked Sep 17, 2026
16Indirect evidenceBridgewater Township, NJ, United StatesHQ checked Sep 17, 20261,001-5,0001,512 contacts →

Indirect 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
17Indirect evidenceWarren, NJ, United StatesHQ checked Sep 17, 2026501-1,0001,212 contacts →

Indirect 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
18Indirect evidenceCovington, KY, United StatesHQ checked Sep 17, 20261,001-5,0001,043 contacts →

Indirect evidence

“CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services…”

Source: RevenueBase Database · checked Sep 17, 2026
19Indirect evidenceLeiden, NetherlandsHQ checked Sep 17, 2026201-500976 contacts →

Indirect evidence

“Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.”

Source: RevenueBase Database · checked Sep 17, 2026
20Partial evidenceStamford, CT, United StatesHQ checked Sep 17, 2026501-1,000885 contacts →

Partial 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
21Indirect evidencePalo Alto, CA, United StatesHQ checked Sep 17, 2026501-1,000837 contacts →

Indirect evidence

“We exist to bring transformative medicines to people living with genetic conditions.”

Source: RevenueBase Database · checked Sep 17, 2026
22Indirect evidencePrinceton, NJ, United StatesHQ checked Sep 17, 2026501-1,000727 contacts →

Indirect 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
23Indirect evidenceLondon, United KingdomHQ checked Sep 17, 2026501-1,000709 contacts →

Indirect evidence

“Partner of choice in specialty, hospital, and rare disease medicines ADVANZ PHARMA is a global pharmaceutical company with the purpose to improve patients’ lives by providing the…”

Source: RevenueBase Database · checked Sep 17, 2026
24Indirect evidencePasadena, CA, United StatesHQ checked Sep 17, 2026501-1,000657 contacts →

Indirect 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
25Indirect evidenceAllschwil, SwitzerlandHQ checked Sep 17, 2026501-1,000644 contacts →

Indirect evidence

“The purpose of Idorsia is to discover, develop, and commercialize innovative medicines to help more patients.”

Source: RevenueBase Database · checked Sep 17, 2026

Showing 25 of 625 orphan drug manufacturers worldwide

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Orphan drug manufacturers worldwide at a glance

As of Sep 17, 2026
40.1%Verified-email coverage
341.4Average contacts per company
11-50Median size (employees)
3.8%Largest-city concentration · London
Sep 17, 2026Page data refreshed

Companies by employee count

1-10
200
11-50
202
51-200
112
201-500
48
501-1,000
25
1,001-5,000
22
5,001-10,000
4
10001+
10

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 orphan drug manufacturers, 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.
Coverage
7 countries, largest first: the United States, India, the United Kingdom, France, Canada, Switzerland, the Netherlands. Country and city lists for orphan drug manufacturers are linked below.
What qualifies
Companies worldwide whose own description of what they do matches “orphan drug manufacturers”. 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 orphan drug manufacturers), 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 orphan drug manufacturers, and records with no verified work email.
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, 85,537 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.

Orphan drug manufacturers by location

7 lists · largest first

Every Orphan drug manufacturers list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.

Countries

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 orphan drug manufacturers, 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 orphan drug manufacturers are there worldwide?

RevenueBase identified 625 orphan drug manufacturers worldwide 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 country has the most orphan drug manufacturers?

The United States leads with 334 companies, followed by India (98) and the United Kingdom (65). Every orphan drug manufacturers list by country and city is linked on this page.

Which city has the most orphan drug manufacturers?

London leads with 24 headquartered companies, followed by Cambridge (15) and Mumbai (15).

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 85,537 verified decision-maker contacts tracked across these 625 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.