Ultragenyx Pharmaceutical Inc. RARE announced positive interim data from a phase II study on UX007 (triheptanoin) for the treatment of patients with long-chain fatty acid oxidation disorder (LC-FAOD). Data were collected at the end of the initial 24-week treatment period.
The single-arm, open-label phase II study evaluated patients with moderate-to-severe FAOD across three symptoms groups – musculoskeletal, liver/hypoglycemia, and cardiac symptoms.
Ultragenyx expects to reveal 78-week data from the phase II study in second half of 2016. The company will provide an update on the design and timing of a phase III study on UX007 in the first half of 2016.
UX007 is also being evaluated in the phase II Glut1 DS seizure study for the treatment of patients with glucose transporter type-1 deficiency syndrome (Glut1 DS). Interim data is expected by the end of 2015 or early 2016.
UX007 has Orphan drug status in the U.S. for the treatment of FAOD and Glut1 DS. In EU, UX007 has Orphan drug status for the treatment of Glut1 DS and Orphan medicinal product designation for very long-chain acyl-coa dehydrogenase (VLCAD) deficiency.
In addition, last month, the European Commission granted Orphan medicinal product designation to three other subtypes of LC-FAOD – long-chain 3-hydroxyacyl-coa dehydrogenase (LCHAD) deficiency, trifunctional protein (TFP) deficiency and carnitine palmitoyltransferase II (CPT-II) deficiency.
Meanwhile, the company continues to progress on its pipeline. Currently, the company is evaluating aceneuramic acid extended release (Ace-ER) in a pivotal phase III study for the treatment of patients with GNE myopathy. Results are expected in early 2017.
We note that earlier in the month, a marketing application was filled for Ace-ER in the EU, based on phase II results for the treatment of adult patients with GNE myopathy. A response from the European Commission is expected in the second half of 2016.
Other companies that focus on the development of products for rare and ultra-rare diseases include BioMarin Pharmaceutical Inc. BMRN, Sarepta Therapeutics, Inc. SRPT and Alexion Pharmaceuticals, Inc. ALXN.
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