The Food and Drug Administration (FDA) has approved the use of Swedish Orphan Biovitrum’s (Sobi’s) orfadin oral suspension for the treatment of Hereditary Tyrosinemia Type 1 (HT-1).
Orfadin is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase that can be used to cure HT-1.
HT-1 is a rare genetic disease affecting infants and children and the ones suffering from HT-1 face problems breaking down an amino acid called tyrosine.
This results in the formation and accumulation of toxic by-products in the body and can cause renal, liver and neurological problems, which can be fatal if untreated.
Orfadin blocks the breakdown of tyrosine and helps reduce the amount of toxic by-products in the body.
Available in a 4mg/mL dose, it should be used in combination with dietary restrictions of tyrosine and phenylalanine.
The new formulation, which is an alternative to the capsules, serves as an additional option for infants and young kids.
Sobi Medical Affairs North America vice-president and head Len Walt said: "We continue to support the HT-1 community with the introduction of the oral suspension.
"This new liquid formulation provides an additional option for parents with small children taking ORFADIN, or for anyone who has difficulty swallowing capsules."
Orfadin oral suspension will be available on the market from August this year.
Based in Stockholm, Sweden, Sobi is an international specialty healthcare company that works on rare diseases.