Sobi Receives Approval From Health Canada for Orfadin (nitisinone) Oral Suspension for the Treatment of HT-1

OAKVILLE, Ontario--(BUSINESS WIRE)-- Sobi, a pioneering international biopharmaceutical company dedicated to rare and difficult-to-treat diseases, has received approval from Health Canada for an oral suspension formulation of Orfadin^® (nitisinone). Orfadin is the first medicine approved in multiple countries globally for use in combination with dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder that may result in liver, renal and neurological complications and in most cases is fatal if untreated.

“Oral suspension Orfadin is good news for parents of infants with HT-1 who typically cannot swallow a capsule, and we are grateful to Sobi for bringing this formulation to Canada. Indeed, Canadians with HT-1 have benefitted from Sobi’s commitment in providing Orfadin capsules for many years through the Special Access Program when there was no other drug available and recently in securing regulatory approval through Health Canada,” said Durhane Wong-Rieger, President of the Canadian Organization for Rare Disorders.

“The introduction of new nitisinone dosing options, such as Orfadin oral suspension, is the result of Sobi listening to feedback from patients and caregivers and continuing to innovate to meet their needs,” said Bob McLay, Vice President and General Manager of Sobi Canada Inc. “In various markets around the world where Sobi has already introduced multiple innovative Orfadin formulations such as Orfadin oral suspension and Orfadin 20mg capsules, we have seen patients increasingly choose these dosing options over the first generation capsule, so we are excited to offer a choice to patients in Canada as well.”

Both Orfadin oral suspension and Orfadin 20mg capsules are available in Canada, as well as the original 2mg, 5mg and 10mg capsules. The original Canadian approval of Orfadin was based on a multinational, uncontrolled, open-label study of 291 HT-1 patients. Patients younger than 2 months of age who were treated with dietary restrictions and Orfadin experienced 2-, 4-, and 6-year survival probabilities of 93%, compared to 2- and 4-year survival probabilities of 38% and 29%, respectively, for historical controls treated with dietary restriction alone. The most common reported adverse reactions in the trial were thrombocytopenia, leucopenia, granulocytopenia and conjunctivitis, corneal opacity, keratitis, and photophobia. No patients discontinued treatment due to adverse drug reactions. Please see important safety information and full prescribing information at the Sobi North America website.

About hereditary tyrosinemia (HT-1)
People with hereditary tyrosinemia 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, renal and neurological complications. Approximately 1,000 persons worldwide are identified as living with HT-1 today. Roughly 10 percent of those patients reside in Canada.

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. 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 U.S. prescribing information, please see www.orfadin.com. Full Canadian prescribing information can be found on the Sobi North America website.

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 www.sobi.com.

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