Orfadin Oral Solution Approved
April 22, 2016 – The U.S. Food and Drug Administration (FDA) approved Orfadin® (nitisinone) in a 4mg/mL oral solution formulation manufactured by Swedish Orphan Biovitrum AB (Sobi™). Used along with diet limitations, Orfadin® treats the rare genetic disease hereditary tyrosinaemia type-1 (HT-1) which affects only a few hundred patients – mainly children. Individuals who have HT-1 cannot utilize tyrosine, an amino acid, leading to the accumulation of impurities in their blood. Orfadin blocks an enzyme to prevent the buildup of tyrosine by-products. Orfadin doses change frequently because they are determined by the patient’s weight. A liquid form of the drug makes both dosing and dose adjustments easier. Previously, Orfadin was available only in 2mg, 5mg and 10mg capsules that had to be mixed with water for children who could not swallow them.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by regular mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- Regular Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
Actual drug patent expiration dates and a availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.