Rare Disease Therapeutics
Rare Disease Therapeutics is a privately held, Franklin, Tennessee-based pharmaceutical commercialization company founded in 1991, specializing in bringing FDA-approved orphan drug treatments to the U.S. market on behalf of pharmaceutical manufacturers and small biotech firms through regulatory, sales, marketing, and distribution services.
- Company typePrivate
- Founded1991
- HeadquartersFranklin, United States
- Headcount1–10
- GTM typeB2B
- OfferingServices
What Rare Disease Therapeutics does
Rare Disease Therapeutics, Inc. (RDT) is a privately held, Franklin, Tennessee-based pharmaceutical commercialization company founded in 1991 as America's first exclusively orphan drug company. The company specializes in bringing treatments for rare diseases (conditions affecting fewer than 200,000 people) to the U.S. market on behalf of pharmaceutical manufacturers and small biotech firms. RDT provides end-to-end commercialization services spanning regulatory affairs (INDs, BLAs, NDAs), targeted sales and marketing, medical affairs, pharmacovigilance, quality assurance, and single-source distribution. Its current product portfolio includes four FDA-approved treatments: ANASCORP and ANAVIP (antivenoms for scorpion stings and pit viper snake bites, respectively), PURIXAN (mercaptopurine oral suspension for acute lymphoblastic leukemia), and Xromi (hydroxyurea oral solution for sickle cell disease), plus an authorized generic of mercaptopurine.
RDT's business model operates on two complementary tracks: direct commercialization of its own FDA-approved rare disease products, and fee-based commercialization partnership services for external pharmaceutical manufacturers seeking U.S. market entry. The company works with three principal partner categories — domestic pharmaceutical manufacturers, small biotech development companies, and international manufacturers seeking U.S. access — offering services from Phase 2/3 clinical trial support through post-approval regulatory maintenance. Distribution is executed through a specialized salesforce that targets healthcare specialists treating rare disease patients and secures contracts with large healthcare systems. RDT holds WBENC Women's Business Enterprise and WOSB Women-Owned Small Business certifications.
The company is led by CEO Caroline Ellis Durst, who succeeded founder Milton Ellis in 2015. RDT has accumulated over 30 years of regulatory experience and has received two National Organization for Rare Disorders (NORD) awards — the 2000 Humanitarian Award for ORFADIN (nitisinone) and the 2012 Partner In Progress Award for ANASCORP. The company operates exclusively in the U.S. market and has no disclosed subsidiaries, parent company, external investors, or funding rounds. Its previous name was Orphan Pharmaceuticals USA, Inc.
Rare Disease Therapeutics firmographics
Firmographics- Name
- Rare Disease Therapeutics
- Legal name
- Rare Disease Therapeutics, Inc.
- Website
- https://raretx.com
- Company type
- Private
- Founded year
- 1991
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Rare Disease Therapeutics is a privately held, Franklin, Tennessee-based pharmaceutical commercialization company founded in 1991, specializing in bringing FDA-approved orphan drug treatments to the U.S. market on behalf of pharmaceutical manufacturers and small biotech firms through regulatory, sales, marketing, and distribution services.
- Ownership category
- akta.pro rank
Rare Disease Therapeutics industry classification
Industry- Product category
- Orphan Drug Commercialization Services
- NAICS
- Pharmaceutical Preparation Manufacturing (325412), Biological Product (except Diagnostic) Manufacturing (325414), Pharmaceutical and Medicine Manufacturing (3254)
- SIC
- Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836), Wholesale-Drugs, Proprietaries & Druggists' Sundries (5122)
- akta.pro primary industry
- Rare Oncology & Hematologic Malignancy Therapies (HLAIAIAC)
- akta.pro secondary industries
- Rare Infectious Disease Therapies (HLAIAIAF), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)
Keywords
Where Rare Disease Therapeutics is headquartered
LocationHeadquarters
- HQ city
- Franklin
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Rare Disease Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Operations, Marketing or Sales, Technology or R&D, Supply Chain
Revenue model
- Commercialization Services: RDT generates revenue by providing comprehensive commercialization services to pharmaceutical manufacturers with orphan drugs in development or approved products requiring regulatory, sales, marketing, and distribution support. Services include clinical trial support, regulatory submissions, targeted sales and marketing, pharmacovigilance, and quality assurance for orphan drugs.
Go-to-market motion2 records
Distribution channels2 records
Marketing channels3 records
Rare Disease Therapeutics product offering
Product offeringCore offering
Rare Disease Therapeutics operates as America's first exclusively orphan drug company, providing comprehensive commercialization services for pharmaceutical manufacturers developing rare disease treatments. The company combines regulatory affairs expertise (INDs, BLAs, NDAs), targeted sales and marketing, medical affairs, pharmacovigilance, and single-source distribution to bring orphan drugs through FDA approval and to patients with rare diseases. Additionally, RDT maintains a portfolio of FDA-approved specialty pharmaceutical products including antivenoms and hematology/oncology treatments.
Product overview
Rare Disease Therapeutics operates as America's first exclusively orphan drug company, offering a portfolio of FDA-approved rare disease treatments along with commercialization services. The product portfolio spans antivenoms (ANASCORP and ANAVIP for scorpion and snake bites), hematology/oncology treatments (PURIXAN/mercaptopurine and Xromi/hydroxyurea), and previously sold products including ORFADIN (nitisinone) for hereditary tyrosinemia type 1 and CYSTADANE (betaine anhydrous) for homocystinuria. The company also provides full-service orphan drug commercialization support including regulatory affairs, clinical trial consulting, sales and marketing, medical affairs, and pharmacovigilance for pharmaceutical manufacturers with orphan drugs in development or approved products requiring regulatory and commercial support.
Differentiator
Problem solved
Functional benefit
Products and services
- ANASCORP ANASCORP [centruroides (scorpion) immune F(ab')2 (equine)] is an antivenom injection for the treatment of scorpion stings, developed using centruroides immune F(ab')2 equine source. FDA-approved product distributed to healthcare systems for treating patients with venomous scorpion stings.
- ANAVIP
Quantifiable outcome
- Transformed treatment of hereditary tyrosinemia type 1 - replaced liver transplantation as first-line treatment with 90% of patients achieving liver failure control
- +1 more outcomes
Companies that use Rare Disease Therapeutics
Customer profileSegments2 records
Ideal customer profiles2 records
Rare Disease Therapeutics technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Rare Disease Therapeutics partnerships and signals
Strategic signalPartnerships
Three partnerships are on record, tiered core.
- Pharmaceutical Manufacturers with Orphan DrugscoreRDT seeks strategic alliances with pharmaceutical manufacturers that have orphan drugs in development or approved products requiring U.S. commercialization support. Services include orphan drug development, clinical development, regulatory support for FDA registration, sales and marketing, and distribution.
- Small Biotech Development CompaniescoreRDT partners with small biotech companies developing orphan drugs to provide commercialization services including regulatory affairs support, targeted sales and marketing, pricing and reimbursement strategies, and single-source distribution to help bring rare disease treatments to market in the U.S.
- International ManufacturerscoreRDT works with international manufacturers to bring rare disease treatments to market in the U.S. by navigating FDA regulatory requirements. This partnership enables global orphan drug manufacturers to access the U.S. market through RDT's commercialization expertise.
Scale indicators2 records
Recent moves6 records
Rare Disease Therapeutics competitors and assessment
Company assessmentMarket position
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
Rare Disease Therapeutics social profiles
Digital presenceRare Disease Therapeutics compliance and trust
Trust signalCompliance3 records
Rare Disease Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Rare Disease Therapeutics leadership team
Management profileNumber of profiles
Profiles1 record
Rare Disease Therapeutics funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Rare Disease Therapeutics M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Rare Disease Therapeutics
What does Rare Disease Therapeutics do?
Rare Disease Therapeutics operates as America's first exclusively orphan drug company, providing comprehensive commercialization services for pharmaceutical manufacturers developing rare disease treatments. The company combines regulatory affairs expertise (INDs, BLAs, NDAs), targeted sales and marketing, medical affairs, pharmacovigilance, and single-source distribution to bring orphan drugs through FDA approval and to patients with rare diseases. Additionally, RDT maintains a portfolio of FDA-approved specialty pharmaceutical products including antivenoms and hematology/oncology treatments.
Is Rare Disease Therapeutics a public or private company?
Rare Disease Therapeutics is a private company. It is classified as family owned and is currently operating.
When was Rare Disease Therapeutics founded?
Rare Disease Therapeutics was founded in 1991. It employs 1 to 10 people.
Where is Rare Disease Therapeutics based?
Rare Disease Therapeutics is headquartered in Franklin, United States, in the North America region.
How does Rare Disease Therapeutics make money?
One revenue line is on record: commercialization Services.
Does Rare Disease Therapeutics have an API?
No public API is recorded for Rare Disease Therapeutics.
What industry is Rare Disease Therapeutics in?
Rare Disease Therapeutics's product category is Orphan Drug Commercialization Services. Its primary akta.pro industry code is HLAIAIAC, Rare Oncology & Hematologic Malignancy Therapies, with a secondary code of HLAIAIAF, Rare Infectious Disease Therapies. Its NAICS code is 325412 and its SIC code is 2834.