FDA validates Orfadin® oral suspension filing

Sobi (Swedish Orphan Biovitrum AB (publ)) today announced that the Food and Drug Administration (FDA) has initiated review of the oral suspension formulation of Orfadin (nitisinone) for the treatment of Hereditary Tyrosinaemia type-1 (HT-1). HT-1 is a rare genetic disease characterised by signs and symptoms that begin in the first few months of life. In affected infants the disease may result in liver and kidney failure, and can be fatal if not diagnosed and treated early in life. 

The dossier was submitted to FDA in June and a formal decision is expected during the second quarter 2016. The oral suspension of Orfadin, if approved,  will facilitate dosing accuracy and will simplify ease of use by reducing the number of steps required to administer the medicine.

"The oral suspension formulation is a demonstration of Sobi's commitment to the needs of the infants and children diagnosed with HT-1 early in life. As new-born screening for HT-1 is becoming more widespread the need for such formulations is evident," says Michael Yeh, Vice President Head of Medical for Sobi in North America. "We are pleased about the FDA validation of the filing of the Orfadin oral suspension, and believe that the oral suspension will contribute to address an unmet need and improve the treatment opportunities of paediatric patients with HT-1."

Orfadin is the only FDA approved therapy for use as an adjunct to dietary restriction of tyrosine and phenylalanine for the treatment of hereditary tyrosinaemia type 1 (HT-1). It is also approved in the EU and several other countries and the European Commission recently approved the oral suspension formulation of Orfadin.

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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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