Pharma Focus Asia

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

Tuesday, November 28, 2017

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.

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