Ultragenyx Secures UK Approval for Triheptanoin's Oral Liquid Formulation to Treat LC-FAOD Rare Inherited Disorders

MT Newswires Live10-30 23:31

Ultragenyx (RARE) has secured the UK Medicines and Healthcare products Regulatory Agency's approval for an oral liquid formulation of triheptanoin, or Dojolvi, to treat adult and paediatric patients with long-chain fatty acid oxidation disorders or LC-FAOD, the regulator said Friday.

The product has a conditional marketing authorization, as further proof of its efficacy and safety from an ongoing paediatric study is required for a full marketing authorization, MHRA said.

Triheptanoin is a liquid that is taken daily by mouth, as part of a carefully controlled diet, according to the statement.

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