Orfadin, an oral suspension which is meant to treat hereditary tyrosinemia type 1 (HT-1), has been approved by Health Canada.
Sobi, the Oakville, Ont.-based specialty biopharmaceutical that manufactures the drug said Ofradin (nilisinone) is the first medicine approved in multiple countries for use in combination with dietary restrictions of tyrosine and phenylalanine in the treatment of HT-1.
HT-1 is a rare genetic disorder that may result in liver, renal and neurological complications and in most cases is fatal if untreated.
People with hereditary tyrosinemia type 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 per cent of those patients live in Canada.
“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,” according to Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders.
Before the Health Canada approval, he said, Sobi had been part of a Special Access Program which allowed Canadians with HT-1 to access Orfadin capsules when there was no other drug available in the country.
“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.”
Orfadin blocks the breakdown of tyrosine. This lowers the number 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.
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.