The COMP issues an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission (EC) for endorsement of the opinion. Orphan drug designation by the EC provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.Background on GSDIa GSDIa, the most common genetically inherited glycogen storage disease, has significant unmet needs. Patients have a defective gene for the enzyme glucose-6-phosphatase-a, resulting in the inability to regulate blood sugar (glucose). Hypoglycemia in GSDIa patients can be life-threatening, while the accumulation of the complex sugar glycogen in certain organs and tissues can impair the ability of these tissues to function normally. If chronically untreated, patients develop severe lactic acidosis, can progress to renal failure, and die in infancy or childhood. There are no approved pharmacological therapies, and no ongoing clinical development beyond dietary approaches. An estimated 6,000 or more patients are affected by GSDIa worldwide. About Dimension Therapeutics, Inc. Dimension Therapeutics, Inc. (NASDAQ:DMTX) is the leader in discovering and developing new therapeutic products for people living with devastating rare and metabolic diseases associated with the liver, based on the most advanced, mammalian adeno-associated virus (AAV) gene delivery technology. Dimension is actively progressing its broad pipeline, which features programs addressing unmet needs for patients suffering from inherited metabolic diseases, including OTC deficiency, GSDIa, citrullinemia type 1, PKU, Wilson disease, a collaboration with Bayer in hemophilia A, and a wholly owned clinical program in hemophilia B. The company targets diseases with readily identifiable patient populations, highly predictive preclinical models, and well-described, and often clinically validated, biomarkers. Founded in 2013, Dimension maintains headquarters in Cambridge, Massachusetts.
For more information, please visit www.dimensiontx.com.Cautionary Note Regarding Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the clinical and developmental progress of Dimension's portfolio and lead programs, including DTX401, the timing, scope, or likelihood of regulatory filings and approvals, the progress and results of preclinical studies, and our ability to develop and advance product candidates into, and successfully complete, clinical studies and the continued development of our manufacturing platform. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Dimension's product candidates, including its candidate, DTX401, will not successfully be developed or commercialized in the times indicated or at all; and the risks described under the caption "Risk Factors" in Dimension Therapeutics' Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which is on file with the Securities and Exchange Commission, as well as other risks detailed in Dimension Therapeutics' additional filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Dimension Therapeutics undertakes no duty to update this information unless required by law.
Contacts:Jean FranchiChief Financial OfficerDimension Therapeutics617firstname.lastname@example.orgBurns McClellan, on behalf of Dimension TherapeuticsMedia: Justin Jackson212-213-0006, email@example.com