What ITF Therapeutics does

ITF Therapeutics LLC is a U.S. specialty pharmaceutical company that develops and commercializes rare disease therapies. Established in January 2024, the company operates as the U.S. rare disease affiliate of Italfarmaco Group, an Italian family-owned pharmaceutical firm founded in 1938 that distributes products across more than 90 countries. ITF Therapeutics is headquartered in Concord, Massachusetts, and maintains a 11–50 person organization focused on commercialization, medical affairs, patient access, and patient advocacy for the U.S. market.

The company's core asset is DUVYZAT (givinostat), an orally administered histone deacetylase (HDAC) inhibitor and the first nonsteroidal therapy approved for Duchenne muscular dystrophy (DMD) in patients aged 6 and older. The mechanism is independent of the specific dystrophin gene mutation, reducing excessive HDAC activity in DMD muscle to decrease inflammation, enhance muscle repair capacity, and slow muscle loss. FDA approval was secured in March 2024 and U.S. commercial launch followed in July 2024, with subsequent approvals from the UK MHRA (December 2024) and the European Commission (June 2025). The clinical evidence base includes the Phase 3 EPIDYS trial and open-label extension data covering up to eight years of treatment, with ten abstracts presented at the 2026 MDA Clinical and Scientific Conference.

ITF Therapeutics generates revenue through prescription product sales of DUVYZAT, supplemented by a pipeline exploring givinostat in Becker muscular dystrophy and polycythemia vera. The company sells through a traditional pharmaceutical field sales model targeting neuromuscular specialists and pediatricians, with prescriptions initiated via a Patient Start Form on duvyzat.com. Patient access is supported by ITF ARC, a comprehensive patient support program offering insurance navigation, financial assistance for eligible patients, and pharmacist support. The go-to-market is reinforced by a dense network of patient advocacy partnerships (MDA, PPMD, Cure Duchenne, Jett Foundation, NORD, Team Joseph, and others) and a leadership team drawn primarily from Genzyme, Biogen, Vertex, and bluebird bio.

Name

ITF Therapeutics

Legal name

ITF Therapeutics LLC

Website

https://itftherapeutics.com

Company type

Private

Founded year

2024

Operating status

Operating

Headcount range

11–50 employees

Short description

ITF Therapeutics is the U.S. rare disease affiliate of Italfarmaco Group that develops and commercializes DUVYZAT (givinostat), an FDA-approved HDAC inhibitor for Duchenne muscular dystrophy patients aged 6 and older, supported by the ITF ARC patient access program.

Ownership category

akta.pro rank

Product category

Rare Disease Pharmaceuticals - Duchenne Muscular Dystrophy

NAICS

Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Cardiometabolic Pharmaceuticals (Cardiovascular/Diabetes/Obesity) (HLAIAAAE)

Keywords

• Rare disease therapeutics
• Duchenne muscular dystrophy treatment
• HDAC inhibitor therapy
• Pharmaceutical commercialization
• Muscular dystrophy drugs

Headquarters

HQ city

Concord

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Marketing or Sales, Supply Chain, Operations

Revenue model

1. Pharmaceutical Product Sales: Revenue generated from commercial sales of FDA-approved DUVYZAT (givinostat) for the treatment of Duchenne muscular dystrophy. DUVYZAT is prescribed to patients 6 years of age and older through healthcare providers. The company also has pipeline products in development including Becker muscular dystrophy and polycythemia vera indications.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels7 records

Core offering

ITF Therapeutics develops and commercializes rare disease pharmaceutical treatments, with its primary product being DUVYZAT (givinostat), an FDA-approved orally administered histone deacetylase (HDAC) inhibitor for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. The company operates as the U.S. affiliate of Italfarmaco Group, leveraging parent-company R&D and pipeline assets for U.S. commercialization. It also offers the ITF ARC patient support program providing insurance navigation, financial assistance, and pharmacist support services to facilitate patient access.

Product overview

ITF Therapeutics operates as the U.S. affiliate of Italfarmaco Group, focused on developing and commercializing rare disease treatments. The company's primary product is DUVYZAT (givinostat), an FDA-approved HDAC inhibitor for Duchenne muscular dystrophy. DUVYZAT is the first and only nonsteroidal therapy approved for DMD that works independently of the specific dystrophin gene mutation. The company also provides ITF ARC (Access, Resources and Care), a patient support services portfolio that includes insurance navigation assistance, personalized pharmacist support, financial assistance, and educational materials. The pipeline includes givinostat for Becker muscular dystrophy and polycythemia vera.

Differentiator

Problem solved

Functional benefit

Brands

• DUVYZAT: FDA-approved histone deacetylase (HDAC) inhibitor for the treatment of Duchenne muscular dystrophy in patients 6 years of age and older. The product was discovered through Italfarmaco's research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy).
• ITF ARC

Products and services

• DUVYZAT (givinostat) DUVYZAT (givinostat) is an FDA-approved orally administered histone deacetylase (HDAC) inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. It is the first and only nonsteroidal therapy approved for DMD that works independently of the patient's specific dystrophin gene mutation, reducing inflammation, increasing muscle repair capacity, and slowing muscle loss.
• ITF ARC (Access, Resources and Care) Patient Support Program ITF ARC is a comprehensive patient support service portfolio offering insurance coverage navigation, personalized pharmacist support, financial and access assistance for eligible patients, and educational materials to facilitate treatment access for patients prescribed DUVYZAT.

Quantifiable outcome

• Delay in loss of ability to rise from floor by median 2.0 years
• +3 more outcomes

Named customers5 records

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

Eleven partnerships are on record, tiered major, core and minor.

• Parent Project Muscular Dystrophy (PPMD)majorStrategic or Co-development Partner · 6 March 2026PPMD partnered with the Cooperative International Neuromuscular Research Group (CINRG) to support the Expanded Duchenne Natural History Study (eDNHS). Multiple pharmaceutical companies, academic institutions, and clinical sites worldwide are involved in the study to gather long-term data on DMD to facilitate therapy development.
• Italfarmaco GroupcoreStrategic or Co-development Partner · 1 January 2024ITF Therapeutics is the US rare disease affiliate of Italfarmaco Group, established in January 2024. Italfarmaco founded in 1938 in Milan, Italy, operates in 90+ countries. ITF leverages Italfarmaco's R&D capabilities and pipeline products for US commercialization.
• Muscular Dystrophy Association (MDA)majorGTM or Marketing PartnerMDA is one of ITF Therapeutics' key patient advocacy partners, collaborating to support families affected by Duchenne muscular dystrophy through sponsorships, workshops, and conferences.
• Cure DuchennemajorGTM or Marketing PartnerNational nonprofit focused on finding treatments and a cure for Duchenne muscular dystrophy. ITF partners with Cure Duchenne to support patient communities.
• Team JosephmajorGTM or Marketing PartnerTeam Joseph supports the Duchenne Family Assistance Program providing personalized guidance, support, and financial assistance to individuals and families affected by DMD, including medical equipment, home modifications, and travel assistance.
• Jett FoundationcoreGTM or Marketing PartnerNonprofit foundation raising awareness, educating and supporting families, advancing medical and scientific knowledge, and advocating for legislative and regulatory change in DMD.
• National Organization for Rare Disorders (NORD)coreGTM or Marketing PartnerNORD is a federation of voluntary health organizations dedicated to helping people with rare diseases. ITF partners with NORD to support rare disease communities.
• EveryLife FoundationcoreGTM or Marketing PartnerFoundation focused on advancing the development of treatments and cures for rare diseases by supporting the community and advocating for policy change.
• Little Hercules FoundationminorGTM or Marketing PartnerNonprofit foundation supporting families affected by Duchenne muscular dystrophy.
• The Akari FoundationminorGTM or Marketing PartnerFoundation providing support and resources for families affected by rare diseases.
• Telethon and Duchenne Parent Project (Italy)coreStrategic or Co-development PartnerDUVYZAT was discovered through Italfarmaco's research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy).

Scale indicators6 records

Recent moves7 records

Expansion highlights6 records

Emerging players

• Solid Biosciences: Solid Biosciences is developing SGT-003 gene therapy for Duchenne muscular dystrophy, positioning as a potential curative competitor in the same patient population as DUVYZAT.
• ReveraGen BioPharma: ReveraGen developed vamorolone (Agamree), a dissociative corticosteroid approved for DMD, representing an alternative non-traditional steroid approach competing with DUVYZAT's nonsteroidal positioning.
• Capricor Therapeutics: Capricor is developing deramiocel (CAP-1002), a cell therapy for Duchenne muscular dystrophy, representing an alternative disease-modifying approach competing for the same DMD patient population.
• Edgewise Therapeutics: Edgewise Therapeutics is developing sevasemten (EDG-5506) for Becker and Duchenne muscular dystrophy, targeting the same neuromuscular indication through a different mechanism, representing future direct competition.

Broad incumbents

• Ultragenyx Pharmaceutical: Ultragenyx is a commercial-stage rare disease company with multiple approved therapies and a field-sales-driven model targeting ultra-rare conditions, comparable in commercial approach to ITF Therapeutics.
• BioMarin Pharmaceutical: BioMarin is a larger established rare disease specialty pharmaceutical company with a broad orphan disease portfolio and commercial infrastructure that serves as a comparator for ITF Therapeutics' rare disease go-to-market model.

Direct peers

• Sarepta Therapeutics: Sarepta is the leading commercial-stage DMD-focused company, with Elevidys gene therapy and exon-skipping drugs (eteplirsen, golodirsen, casimersen). It directly competes with ITF Therapeutics for DMD prescriber share and patient access.
• Catalyst Pharmaceuticals: Catalyst is a rare disease specialty pharmaceutical company commercializing therapies through field sales and patient support programs, sharing the orphan-disease commercialization model ITF Therapeutics employs for DUVYZAT.
• PTC Therapeutics: PTC Therapeutics markets Translarna (ataluren) for nonsense mutation DMD outside the U.S. and has an established rare disease commercial franchise overlapping with ITF's neuromuscular focus.

Regional players

• Recordati Rare Diseases: Recordati Rare Diseases is the rare disease arm of Italian-based Recordati Group, operating globally with a portfolio of orphan therapies. As a fellow Italian-headquartered rare disease company with international commercial reach, it parallels the Italfarmaco-ITF model.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat7 records

Key risks5 records

Key highlights7 records

Customer concentration

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Revenue estimate

Valuation estimate

Number of profiles

Profiles9 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 ITF Therapeutics