Sobi says US FDA approves higher dose of Orfadin to treat rare genetic disorder

pharmafile | June 15, 2016 | News story | Research and Development, Sales and Marketing US FDA, genetic disorder, regulation, sobi 

Swedish Orphan Biovitrum AB (Sobi) on Wednesday said the US Food and Drug Administration (FDA) has approved a higher strength capsule of Orfadin (nitisinone) to treat a rare genetic disease in infants and children.

Hereditary Tyrosinaemia type-1 is progressive and may result in liver and kidney complications and can be fatal if untreated.

As dosing is adjusted by weight, patients need progressively higher doses as they grow. Sobi has developed the higher capsule strength of 20 mg to support the treatment regimens of adolescent and adult patients that may allow for fewer capsules per dose, the company said in a statement.

This is important because of the chronic nature of their treatment.

Michael Yeh, head of global medical affairs core products at Sobi, said: “The HT-1 patient journey has driven Sobi’s development of a new strength for Orfadin in order to support sustainable outcomes over a lifetime for HT-1 patients.”

Orfadin is approved in the US 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.

Anjali Shukla

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