Orphan Therapeutics

What Orphan Therapeutics does

Orphan Therapeutics, LLC is a privately held drug development company founded in 2003 and headquartered in Longboat Key, Florida. Its foundational program was terlipressin for Hepatorenal Syndrome type 1, whose NDA and development rights were licensed to Ikaria Inc. in March 2010 (Ikaria was acquired by Mallinckrodt in April 2015) and which received FDA approval in September 2022 as Terlivaz (terlipressin) Injection. Orphan Therapeutics' own economic role in Terlivaz is not disclosed, and the company's residual activity appears to be supporting the program's continued regulatory and commercial positioning rather than direct commercialization.

The company has since repositioned around the Orphan Therapeutics Accelerator (OTXL), a tax-exempt nonprofit structure that acquires, funds, and completes development of shelved cell and gene therapy programs for ultra-rare diseases. OTXL applies an AI-based infrastructure to profile and risk-score shelved assets and match them with new clinical sponsors, CROs, and CDMOs, and has independently evaluated more than 80 shelved rare disease programs over the past two years. In December 2025, OTXL signed an MoU with Fondazione Telethon to commercialize Waskyra, an FDA-approved ex vivo gene therapy for Wiskott-Aldrich syndrome, in the US.

In 2026, OTXL expanded its ecosystem through two major moves: in February it onboarded UAE-based P4ML as a founding member to support global access pathways, and in May it jointly launched CGTxchange with the American Society of Gene & Cell Therapy (ASGCT), a marketplace that uses OTXL's AI profiling infrastructure to connect shelved CGT programs worldwide with funders and development partners. The company employs 1-10 people, is led by Founder & President Peter Teuber, Ph.D., and discloses no institutional investors, funding rounds, or revenue.

Name

Orphan Therapeutics

Legal name

Orphan Therapeutics, LLC

Website

https://orphantherapeutics.com

Company type

Private

Founded year

2003

Operating status

Operating

Headcount range

1–10 employees

Short description

Orphan Therapeutics is a privately held drug development company that originated terlipressin (FDA-approved as Terlivaz in 2022) and now operates the OTXL nonprofit accelerator and the CGTxchange marketplace, using AI-based profiling to reactivate shelved cell and gene therapies for ultra-rare diseases.

Ownership category

akta.pro rank

Product category

Rare Disease Drug Development

NAICS

Medical and Diagnostic Laboratories (62151)

SIC

Services-Medical Laboratories (8071)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Orphan drug development
• Rare disease therapies
• Specialty pharmaceuticals
• Cell and gene therapy
• Pharmaceutical licensing

Headquarters

HQ city

Longboat Key

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations

Core offering

Orphan Therapeutics is a privately held drug development company that develops and seeks regulatory approval for treatments for rare diseases. The company's primary accomplishment is the development of terlipressin (Terlivaz) for Hepatorenal Syndrome type 1, which received FDA approval in September 2022 after being licensed to Ikaria (later acquired by Mallinckrodt). Through the Orphan Therapeutics Accelerator (OTXL), the company profiles and reactivates shelved cell and gene therapy programs via AI-based infrastructure and the CGTxchange marketplace.

Product overview

Orphan Therapeutics is a privately held drug development company founded in 2003, focused on developing and seeking regulatory approval for treatments for rare diseases. The company's primary product is Terlivaz (terlipressin) Injection, which received FDA approval in September 2022 for the treatment of Hepatorenal Syndrome (HRS) type 1. Orphan Therapeutics operated as a development-stage company, licensing its development rights to Ikaria (subsequently acquired by Mallinckrodt) while continuing to support the FDA approval process. The company is headquartered in Longboat Key, Florida, USA.

Differentiator

Problem solved

Functional benefit

Products and services

• Terlivaz (terlipressin) Injection

Segments1 record

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

Three partnerships are on record, tiered core.

• American Society of Gene & Cell Therapy (ASGCT)coreStrategic or Co-development Partner · 14 May 2026ASGCT and OTXL launched CGTxchange, a joint venture marketplace designed to reactivate shelved cell and gene therapy programs by connecting them with funders and partners. The platform is jointly owned and uses OTXL's AI-based infrastructure. ASGCT is a leading professional organization in the field.
• P4MLcoreStrategic or Co-development Partner · 25 February 2026P4ML, a UAE-based health innovation company, became a founding member of the Orphan Therapeutics Accelerator (OTXL). The partnership supports expanding global development and access pathways for ultra-rare disease treatments amid regulatory changes. P4ML leverages its regional expertise and scientific partnerships to enhance OTXL's mission.
• Fondazione TelethoncoreStrategic or Co-development Partner · 15 December 2025Fondazione Telethon and Orphan Therapeutics Accelerator signed an MoU to commercialize an approved ex vivo gene therapy for Wiskott-Aldrich syndrome (Waskyra) in the US through a non-profit collaboration. Waskyra received FDA approval in December 2025 as the first gene therapy from a nonprofit sponsor. The partnership aims to establish a sustainable market access pathway for ultra-rare disease therapies using a mission-aligned model.

Scale indicators2 records

Recent moves5 records

Expansion highlights5 records

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease therapeutics company with multiple approved products for ultra-rare conditions. Directly comparable to Orphan Therapeutics' founding mission of developing treatments for rare diseases and similar commercial-stage experience in orphan drug development.
• Ultragenyx Pharmaceutical: Commercial-stage biotech focused on rare and ultra-rare genetic diseases, including multiple gene therapy and biologic programs. Comparable to OTXL's focus on gene therapy for rare/monogenic diseases (Waskyra for Wiskott-Aldrich syndrome).
• Sarepta Therapeutics: Commercial-stage gene therapy company focused on rare neuromuscular diseases. Comparable to OTXL's mission around gene therapy for rare diseases, though at significantly larger scale with multiple approved products.
• Alexion (AstraZeneca Rare Disease): Established rare disease therapeutics unit of AstraZeneca focused on ultra-rare conditions. Comparable to OTXL's orphan drug development heritage, particularly relevant as a potential acquirer or commercialization partner for ultra-rare assets reactivated through the OTXL platform.

Direct peers

• Taysha Gene Therapies: Clinical-stage gene therapy company focused on monogenic diseases of the central nervous system. Directly comparable to OTXL's Waskyra partnership model of advancing gene therapies for rare/monogenic disorders using partnership-driven development.
• REGENXBIO: Gene therapy company developing AAV-based treatments for rare diseases, with multiple clinical-stage programs. Comparable to OTXL's gene therapy focus, particularly the cell and gene therapy assets being profiled and reactivated through the CGTxchange platform.
• Passage Bio: Clinical-stage gene therapy company developing AAV-delivered therapeutics for rare monogenic CNS disorders. Comparable to OTXL's mission of advancing gene therapies for ultra-rare diseases through partnership and reactivation of stalled programs.
• Solid Biosciences: Clinical-stage gene therapy company developing treatments for rare genetic diseases including Duchenne muscular dystrophy. Comparable to OTXL's focus on rare/monogenic disease gene therapies, particularly in the niche of programs requiring creative development and commercialization approaches.

Emerging players

• Cure Rare Disease: Nonprofit biotechnology organization developing genetic medicines for ultra-rare diseases. Comparable to OTXL's nonprofit OTXL accelerator model and the Telethon-style nonprofit-sponsored gene therapy pathway exemplified by Waskyra.
• Vigil Neuroscience: Clinical-stage company developing therapeutics for rare neurodegenerative diseases, including microglial-based approaches. Comparable to OTXL's mission of reactivating shelved rare disease programs and developing treatments for ultra-rare neurological conditions.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks2 records

Key highlights6 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Orphan Therapeutics