Sobi Launches Orfadin® (nitisinone) Ambassador Program to Support Patients and Caregivers Living with Hereditary Tyrosinemia Type 1 (HT-1)

- Program to provide peer-to-peer education within the HT-1 community -

Waltham, Mass., Dec. 13, 2017— Sobi, a pioneering international biopharmaceutical company dedicated to rare and difficult-to-treat diseases, is proud to introduce the Orfadin Ambassador program, a peer-to-peer education program within the hereditary tyrosinemia type 1 (HT-1) community.

HT-1 is a rare genetic disorder that may result in liver, renal and neurological complications. The Orfadin Ambassadors are patients and caregivers who can share their experiences living with or managing HT-1, provide guidance in navigating diet and lifestyle challenges, and answer questions about their experiences with Orfadin® (nitisinone). Orfadin is the first medicine approved for the treatment of HT-1; it must be used in combination with dietary restriction of tyrosine and phenylalanine; please see important safety information below.

“When the doctor told us our daughter Claire had HT-1, we were so scared and had no idea what to expect,” said Shannon, a mother and Orfadin Ambassador. Over time, Shannon has learned ways to educate her local school and community about Claire’s needs, and how to plan ahead to manage Claire’s HT-1. “We keep learning new ways to take care of Claire. Having a supportive community that understands what you are going through is invaluable. I hope that by sharing my story, I can make the journey a little easier for other families living with this condition.”

“Just because you have HT-1 doesn’t mean you can’t do most of the things you want to do. But you have to take care of yourself, follow your diet, and take your medicine as prescribed by your physician,” said Colin, a 22-year-old skier and videographer, who is also an Orfadin Ambassador.

The Orfadin Ambassador program is a phone-based, peer-to-peer program that allows adults with HT-1 or caregivers of HT-1 patients to talk to another patient or caregiver who has experience with HT-1 and Orfadin. The goal of the program is to provide members of the HT-1 community with support and help navigating living with or caring for a person with HT-1. Orfadin ambassadors are paired with a Health Educator on each call; neither may provide medical advice. The Orfadin Ambassador program is supported by Sobi. More information is available at, by calling 844-581-5872, or through

“Sobi is proud to have reliably provided Orfadin to HT-1 patients for almost 25 years, and we are committed to supporting the HT-1 community,” said Rami Levin, President of Sobi in North America. “In addition to providing innovative treatments for HT-1, we listen and strive to address the needs of patients and caregivers, which is why we developed the Orfadin Ambassador program.”

Indication and Usage

Orfadin is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase indicated for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1). 

Important Safety Information

  • Tyrosine levels can increase in the blood if you do not restrict tyrosine and phenylalanine in your diet while taking Orfadin. Too much tyrosine in the blood can cause serious eye problems or other complications

  • Do not adjust your Orfadin dosage in order to lower the tyrosine levels in the blood. 

  • A reduction in the number of white cells and platelets in the blood have been observed during treatment with Orfadin. Your platelet and white blood cell counts should be monitored regularly during Orfadin treatment.

  • The most common adverse reactions to taking Orfadin are liver cancer, liver failure, low platelets or white cells in the blood, and complaints related to the eyes, including conjunctivitis, corneal opacity, inflammation of the cornea, and extreme sensitivity to light.

  • Tell your physician promptly if you have unexplained eye symptoms, rash, jaundice (yellowing of the skin or whites of the eyes) or excessive bleeding.

  • Use Orfadin during pregnancy only if the potential benefit justifies the potential risk to the fetus.

  • Nursing women should discontinue either Orfadin or breast-feeding based on the recommendation of your healthcare professional.

For full prescribing information, please visit

About hereditary tyrosinemia (HT-1)

Hereditary tyrosinemia type 1 (HT-1) is an extremely rare but treatable hereditary disorder. When a child has HT-1, their body lacks the enzymes needed to break down the amino acid tyrosine, which comes from food. High levels of tyrosine can build up in the blood and form toxic substances in the liver, kidneys and central nervous system, which can cause liver, renal and neurological complications. Approximately 1,000 persons worldwide are identified as living with HT-1 today.

About Orfadin®
Orfadin® (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic tyrosine by-products in the body. Patients must maintain a special diet in combination with Orfadin treatment as tyrosine is not adequately broken down. Before the introduction of Orfadin, fewer than one-third of infants diagnosed with HT-1 before two months of age lived past their second birthday. Today, many HT-1 patients have entered adolescence or adulthood. Orfadin is a proprietary product and is developed and made available globally by Sobi. For full European prescribing information, please visit the EMA website. For full US prescribing information, please see Full Canadian prescribing information can be found on the Sobi North America website.

Orfadin is the first nitisinone product approved in the U.S. and Europe. The U.S. approval of the once-daily formulation was based on the results of a clinical study in 16 HT-1 patients, comparing a 4-week once-daily and 4-week twice daily dosing regimen. The study showed comparable blood-levels of nitisinone for these two regimens. The original approval in the U.S. in 2002 was based on an open-label study of 207 HT-1 patients, in which patients younger than 2 months of age who were treated with dietary restrictions and Orfadin experienced 2- and 4-year survival probabilities of 88% and 88%, respectively. Data from historical controls showed that patients treated with dietary restriction alone had 2- and 4-year survival probabilities of 29% and 29%, respectively. For patients presenting between 2 and 6 months of age who were treated with dietary restrictions and Orfadin, 2- and 4-year survival probabilities were 94% and 94%, respectively. Data for historical controls showed that patients treated with dietary restriction alone had 2- and 4-year survival probabilities of 74% and 60%, respectively. The most common adverse reactions (incidence >2%) seen with Orfadin use are hepatic neoplasm, liver failure, thrombocytopenia, leucopenia, visual system complaints including conjunctivitis, corneal opacity, keratitis, and photophobia. Please see important safety information below. 

About Sobi™
Sobi is an international specialty healthcare company dedicated to rare diseases. Sobi’s mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on Haemophilia, Inflammation and Genetic diseases. Sobi also markets a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2016, Sobi had total revenues of SEK 5.2 billion (USD 608 M) and about 760 employees. The share (STO: SOBI) is listed on Nasdaq Stockholm. More information is available at   

For more information please contact:

Trista Morrison



Related links