Patients for Bitopertin

What Patients for Bitopertin does

Patients for Bitopertin is a patient-driven advocacy organization headquartered at 7315 Wisconsin Avenue, Suite 400W, Bethesda, Maryland, operating under the umbrella of the United Porphyrias Association via the EPP/XLP Committee for the Approval of Bitopertin. Its sole operational objective is securing FDA approval of Bitopertin, an investigational oral, disease-modifying treatment for Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP) being developed by Disc Medicine. The organization serves the EPP/XLP patient community — patients, families, and advocates living with rare photodermatoses that cause severe burning pain within minutes of sunlight exposure, lifelong isolation, anxiety, depression, and in some cases liver complications — with particular emphasis on pediatric and adolescent patients who currently have no approved treatment options.

Name

Patients for Bitopertin

Legal name

Patients for Bitopertin

Website

https://patientsforbitopertin.org

Company type

Private

Operating status

Operating

Short description

Patients for Bitopertin is a nonprofit patient advocacy organization that campaigns for FDA approval of Bitopertin, an oral investigational treatment for EPP and XLP being developed by Disc Medicine, serving patients, families, and advocates affected by these rare sunlight-induced disorders.

Ownership category

akta.pro rank

Product category

Patient Advocacy Services

NAICS

Individual and Family Services (6241)

SIC

Services-Health Services (8000)

akta.pro industry

PRIMARYHLACAKAF

Medication Adherence, Reminders & Patient Support

Healthcare & Life Sciences/Digital Health & Health IT/ePrescribing, Medication Management & Pharmacy IT

SECONDARYHLACANAO

DTx/SaMD Deployment, Integration & Reimbursement Enablement

Healthcare & Life Sciences/Digital Health & Health IT/Digital Therapeutics (DTx) & Software as a Medical Device (SaMD)

Keywords

• Patient advocacy services
• Rare disease advocacy
• FDA approval campaigns
• Drug access petitions
• Porphyria community outreach

Headquarters

HQ city

Bethesda

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Operations, Marketing or Sales, Personnel, Others

Marketing channels4 records

Core offering

Patients for Bitopertin is a patient-driven advocacy organization that campaigns for FDA approval of Bitopertin, an oral disease-modifying drug treatment developed by Disc Medicine for Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). The organization collects and publishes patient stories, runs petition drives, conducts legislative outreach to members of Congress, and organizes direct contact campaigns with the FDA in support of Bitopertin access. It is led by the EPP/XLP Committee for the Approval of Bitopertin and operates as an advocacy effort under the United Porphyrias Association.

Product overview

Patients for Bitopertin is a patient-driven advocacy organization, not a product company. The organization advocates for FDA approval of Bitopertin, an oral drug treatment developed by Disc Medicine for Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). Bitopertin is undergoing Phase 3 clinical trials (APOLLO study with Helios extension) and is not yet approved by the FDA.

Differentiator

Problem solved

Functional benefit

Segments1 record

Ideal customer profiles1 record

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

Three partnerships are on record, tiered core and minor.

• United Porphyrias AssociationcoreOthersPatients for Bitopertin is a patient-driven effort led by the EPP/XLP Committee for the Approval of Bitopertin under United Porphyrias Association. The organization shares office space at 7315 Wisconsin Avenue, Suite 400W, Bethesda, MD. Kristen Wheeden at United Porphyria Association helped connect patients with Bitopertin clinical trials.
• Disc MedicinecoreOthersDisc Medicine is the pharmaceutical company developing Bitopertin for EPP/XLP treatment. Patients for Bitopertin advocates for FDA approval of Disc Medicine's Bitopertin. The organization supports patients participating in Disc Medicine's Phase 3 APOLLO clinical trial and Helios extension study.
• Clinuvel PharmaceuticalsminorOthersClinuvel Pharmaceuticals manufactures Scenesse (afamelanotide), an FDA-approved implant treatment for EPP (approved in 2019). Some patients mention Scenesse as an alternative treatment while advocating for Bitopertin as an oral option that could serve younger patients and be less expensive.

Recent moves6 records

Expansion highlights4 records

Emerging players

• Global Genes: Alliance of rare disease patient advocacy groups that provides toolkits, training, and amplification for single-disease advocacy organizations like Patients for Bitopertin. Directly comparable in mission and operating model.
• EveryLife Foundation for Rare Diseases: Policy-focused rare disease advocacy nonprofit that runs Capitol Hill advocacy days and Congressional Caucus for Rare Diseases — analogous to the legislative outreach function Patients for Bitopertin performs at a smaller scale.
• National Organization for Rare Disorders (NORD): Umbrella rare disease advocacy organization that runs FDA contact campaigns, legislative outreach, and patient story collection — the same playbook used by Patients for Bitopertin, but at a portfolio level across 7,000+ rare diseases.

Others

• Clinuvel Pharmaceuticals: Manufacturer of Scenesse (afamelanotide), the only currently FDA-approved EPP treatment. Ecosystem participant rather than peer, but shapes the regulatory and competitive landscape in which Patients for Bitopertin operates.
• Disc Medicine: The developer of Bitopertin and the clinical trial sponsor (Phase 3 APOLLO study, Helios extension). Not a peer in the advocacy sense but a tightly coupled ecosystem participant whose regulatory and clinical progress directly determines the advocacy organization's value.

Regional players

• EURORDIS - Rare Diseases Europe: European rare disease patient advocacy umbrella that operates the same petition-to-regulator model across EU markets. Comparable in advocacy mechanics but serves a different geography than the US-focused Patients for Bitopertin.
• American Porphyria Foundation: Separate porphyria-focused patient advocacy organization that historically supported the broader porphyria community including EPP patients. Comparable mission and disease focus, with some overlap in the EPP/XLP constituency.

Direct peers

• United Porphyrias Association: Parent organization and same-disease advocacy group based in Bethesda, MD. Directly comparable as a nonprofit patient advocacy organization focused on the EPP/XLP community and broader porphyrias, sharing office space and leadership with Patients for Bitopertin.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights5 records

Customer concentration

• Instagraminstagram.com/p/DWWnLRcGRqq
• Facebookfacebook.com/1703777381003292
• LinkedInlinkedin.com/company/patients-for-bitopertin

Revenue estimate

Valuation estimate

Number of profiles

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 Patients for Bitopertin