Ultragenyx: FDA Grants Fast Track Designation to Gene Therapy Program

Ultragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the first patient has been dosed in the Phase 1/2 study of DTX401, an adeno-associated virus vector based gene therapy for the treatment of patients with glycogen storage disease type Ia (GSDIa). Data from the three-patient first dose cohort are expected in the second half of 2018.

“For over 20 years, our team and the team at the National Institutes of Health (NIH) have worked for this day to occur.  We are thrilled to see the gene therapy trial for GSD type Ia beginning,” said David Weinstein, M.D., M.M.Sc., Professor and Director, Glycogen Storage Disease Program at Connecticut Children’s Medical Center and UConn Health. “I am confident that patients with this condition around the world could benefit from this trial along with children and adults with other genetic liver diseases.  This is truly an exciting day for the GSD community.”

The U.S. Food and Drug Administration (FDA) has also granted Fast Track designation to DTX401 for the treatment of GSDIa. The FDA Fast Track program is designed to facilitate the development and expedite the review of drugs that are intended to treat serious conditions and fill an unmet medical need. Fast Track designation allows for more frequent interaction with the FDA review team. It also enables eligibility for priority review if relevant criteria are met and the potential for a rolling review of the Investigational New Drug application (IND) as data become available.

“Advancing DTX401 into the clinic through our collaboration with Dr. Weinstein, the NIHand other GSD1a treatment centers marks an important milestone in developing a much-needed new potential treatment for patients with GSDIa, a highly debilitating disease with no approved treatment options,” said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. “As our second inborn error of metabolism gene therapy program enters the clinic, we share the excitement of the GSDIa community. We believe that gene therapy offers the best treatment approach for the development of a new therapy that could profoundly improve the lives of patients living with GSDIa.”

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