Sobi celebrates 25 years with Orfadin® (nitisinone) - a result of ground-breaking Swedish research

To acknowledge that it is 25 years since the first person with the rare genetic disease Hereditary Tyrosinaemia type 1 (HT-1) was treated in clinical trials with Orfadin® (nitisinone), Swedish Orphan Biovitrum AB (publ) (Sobi) (STO: SOBI)  hosted a satellite symposium in Lyon, France in conjunction with the annual SSIEM symposium, Society for the Study of Inborn Errors of Metabolism. HT-1 is a progressive disease that affects infants and children, may result in liver and kidney failure and can be fatal if it is not diagnosed and treated early in life.

The symposium honoured Professor Elisabeth Holme and Professor Sven Lindstedt, two Swedish pioneers in the research of HT-1, both of whom passed away earlier this year. Professor Lindstedt led a group at University of Gothenburg who developed and presented ground-breaking research into HT-1. He and his group established the primary aetiology behind the disease, which led to the discovery of nitisinone. Today Orfadin® (nitisinone) is first line therapy, having replaced liver transplantation, as standard care.

"Thanks to these visionary individuals and to the conduct of research that led to the development of nitisinone, people with HT-1 who 25 years ago had short life expectancy as a consequence of the natural course of the disease, have the possibility of therapeutic intervention that may even allow them to become grandparents", says Birgitte Volck, Chief Medical Officer of Sobi.

Before pharmacological treatment was available, less than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday.^[1] Treatment with Orfadin, combined with dietary restriction of tyrosine and phenylalanine; and more widespread new-born screening leading to early diagnosis have dramatically improved the outcomes for HT-1 patients.^[2] 

Orfadin is approved in the EU, USA and several other countries for the treatment of patients with confirmed diagnosis of hereditary tyrosinaemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. HT-1 is a progressive disease where the body lacks the ability to break down the amino acid tyrosine. Without treatment, HT-1 is ultimately fatal. Orfadin, together with the appropriate diet, is an essential part of effective HT-1 treatment. 

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About Orfadin
Orfadin (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic by-products in the body. People with Hereditary Tyrosinaemia type-1 (HT-1) have problems breaking down an amino acid called tyrosine. Toxic by-products are formed and accumulate in the body, which can cause liver failure, renal dysfunction and neurological complications. In the most common form of the disease, symptoms arise within the first six months of the child's life. Patients must maintain a special diet in combination with Orfadin treatment as tyrosine remains in the body. Approximately 1,000 persons are identified as living with HT-1 today. Orfadin is a proprietary product and is developed by and marketed globally by Sobi.

About Sobi
Sobi is an international specialty healthcare company dedicated to rare diseases. Our 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. We also market a portfolio of specialty and rare disease products for partner companies across Europe, the Middle East, North Africa and Russia. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2014, Sobi had total revenues of SEK 2.6 billion (USD 380 M) and about 600 employees. The share (STO: SOBI) is listed on Nasdaq OMX Stockholm. More information is available at www.sobi.com.

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^[1] van Spronsen FJ, Thomasse Y, Smit GP, et al. Hepatology. 1994; 20(5):1187-1191

^[2] Orfadin EPAR: Product information 25/07/2015 Orfadin -EMEA/H/C/000555 -IB/004