Dimension Therapeutics Receives Positive Opinion for European Orphan Drug Designation for DTX301 for the Treatment of Ornithine Transcarbamylase Deficiency

Dimension Therapeutics, Inc. (NASDAQ:DMTX) announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending DTX301 for designation as an orphan medicinal product for the treatment of Ornithine Transcarbamylase (OTC) Deficiency.
According to the news:

Designed to address the underlying genetic defect, DTX301 delivers stable expression and activity of OTC. DTX301 has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism, which if elevated, can cause serious neurological deficiencies in patients. Dimension has completed candidate selection in its DTX301 program and plans to submit an investigational new drug (IND) application with the FDA in the second half of 2016.

Annalisa Jenkins, MBBS, MRCP, Chief Executive Officer of Dimension stated:

The COMP positive opinion for DX301 in the EU, as well as recent orphan drug designation in the US, underscores the compelling need for new treatments for patients living with OTC deficiency. Current medical options are not curative and unfortunately do not eliminate the risk of metabolic crises from OTC deficiency. The DTX301 approach is designed to address these challenges and offer a new option with improved outcomes for patients. We are making significant progress on growing and advancing our unique pipeline of programs addressing the inherited metabolic diseases associated with the liver. The planned submission of the IND for DTX301 later this year will be another important milestone for our company and our goal to transform the care of patients living with these devastating diseases.

Click here to view the full press release.