Orfadin

Indicated For

Hereditary Tyrosinemia Type 1, HT-1

About This Medication

# Orfadin4U Patient Guide: How to Get Orfadin at Low or No Cost Orfadin4U is Sobi's patient support program offering as little as $0 copay for eligible commercially insured patients taking **Orfadin (nitisinone)** for hereditary tyrosinemia type 1 (HT-1), with additional financial support options for uninsured patients.[1][6] ## About Orfadin and HT-1 **Orfadin (nitisinone)** is the first FDA-approved treatment for HT-1, a rare genetic disease affecting about 1,000 people worldwide, primarily infants and children.[8][9] HT-1 disrupts the body's ability to break down tyrosine and phenylalanine, leading to toxic buildup that can cause severe liver, kidney damage, and even death if untreated.[9] Orfadin works by blocking an enzyme (4-hydroxyphenylpyruvate dioxygenase), preventing harmful substance formation when combined with a strict low-tyrosine/phenylalanine diet.[9] Available as capsules (2mg, 5mg, 10mg, 20mg) or oral suspension (4mg/mL), it's taken daily lifelong.[9][10] Sobi, the manufacturer, has supported HT-1 patients for over 25 years, providing not just medication but holistic care through Orfadin4U.[1][5] This includes pharmacy services, insurance navigation, recipe ideas, lifestyle support, and a peer mentorship program called Orfadin Ambassador.[1][3] ## Who Qualifies for Orfadin4U? Orfadin4U primarily targets **commercially insured patients** with a valid Orfadin prescription for HT-1.[1][6] Eligible patients can receive up to **$10,000 annual copay assistance per patient**, reducing out-of-pocket costs to $0.[1][6] No specific income thresholds are listed for copay help—it's not needs-based for commercial insurance.[6] **Uninsured patients** may qualify for Sobi's Patient Assistance Program (PAP), requiring US citizenship/legal residency, a medically appropriate HT-1 diagnosis, uninsured status, and undisclosed income limits.[2] Proof of income and lack of insurance is needed.[2] Both programs ensure access for eligible US residents (including Puerto Rico).[6] ## Income Eligibility Breakdown Income details vary by track: | Eligibility Track | Income Threshold | Key Notes | |-------------------|------------------|-----------| | **Commercial Copay Assistance** | None specified | Up to $10,000/year; commercially insured only; no needs test.[1][6] | | **Uninsured PAP** | Undisclosed (FPL-based?) | US citizen/legal; proof of income required; diagnosis verified.[2] | | **Individual/Couple/Family** | Not published | Contact program for personalized assessment.[2] | Specific Federal Poverty Level (FPL) percentages aren't detailed publicly—call for details.[2] ## Insurance Requirements - **Commercially insured**: Primary focus—program verifies benefits and covers copays.[6] - **Uninsured**: Separate PAP track with income verification.[2][6] - **Medicare/Government insurance**: Typically ineligible for copay help; explore PAP or other state programs.[6] Orfadin4U handles insurance hurdles, ensuring medication reach via direct ship or doctor's office.[1][6] ## Step-by-Step Application Process 1. **Get Prescribed**: Confirm HT-1 diagnosis; doctor writes Orfadin prescription.[2][8] 2. **Contact Orfadin4U**: Call **(800) 496-6111** or **(877) 473-3179** (7am-6pm CST) to request forms faxed to your doctor's office.[2][6] 3. **Complete Enrollment**: Doctor completes/signs medical section + attaches prescription. Patient signs, adds proof of income/insurance (if applicable).[2][6] 4. **Fax Forms**: Send to **(877) 473-3049** or as instructed.[2][6] 5. **Verification**: Program checks insurance/income (quick for copay).[6] 6. **Approval**: Medication ships to patient or doctor's office.[6] Multiple methods: phone, fax, email (orfadin.us@sobi.com).[6] Doctor enrollment preferred.[6] ## Timeline and Delivery Processing time isn't specified but aims for efficiency—insurance verification precedes shipping.[6] Once approved, meds ship directly to you or your physician's office, "as prescribed."[2][6] Copay cards activate immediately for pharmacy use; no expiration while on Orfadin.[6] Refills auto via ongoing enrollment—no reapplication needed.[6] ## Alternatives if Denied or Other Options - **Nityr (nitisinone)**: FDA-approved biosimilar alternative with potentially different assistance programs—discuss with doctor.[program data] - **Appeal Denial**: Contact program for reasons (e.g., insurance mismatch); resubmit docs.[2] - **Other Resources**: State programs, HT-1 foundations, or Sobi's ambassador peer support (844-581-5872).[3] - **Copay Cards**: General savings sites if ineligible.[6] ## Important Disclaimer This guide summarizes publicly available info as of 2026—program terms change.[1][2][6] Not medical/financial advice. Consult your doctor, pharmacist, or call **(800) 496-6111** for personalized eligibility. Sobi may modify benefits anytime. Eligibility not guaranteed; void where prohibited.

Program information last verified: March 30, 2026