FDA Grants Fast Track Designation for Synlogic’s Treatment of Homocystinuria

The U.S. Food and Drug Administration has granted Fast Track designation to Synlogic’s SYNB1353 for the potential treatment of homocystinuria.

Photo: Aoife Brennan, president and CEO of Synlogic

Homocystinuria (HCU) is a group of rare inherited disorders in which the body is unable to process certain amino acids properly. It is characterized by high levels of homocysteine in the blood and urine, which can lead to thromboembolism, lens dislocation, skeletal abnormalities, developmental delay, and intellectual disability. Treatment options for HCU are currently limited due to efficacy and tolerability.

SYNB1353, an orally administered, non-systemically absorbed drug candidate designed to consume methionine in order to lower homocysteine levels, is currently being evaluated in a phase 1 study in healthy volunteers. It is an engineered strain of the probiotic bacteria E. coli Nissle, which consumes methionine within the gastrointestinal tract, preventing methionine absorption and conversion to homocysteine in plasma.

It is the first drug candidate developed through a research collaboration between Synlogic and Ginkgo Bioworks and the first investigational medicine developed on Ginkgo’s platform to enter the clinic. In 2021, Synlogic shared preclinical data for SYNB1353 that demonstrated the lowering of blood homocysteine levels in non-human primates and mouse models. Synlogic expects to share results from the phase 1 study of SYNB1353 in healthy volunteers by the end of 2022.

The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. To qualify, available clinical and non-clinical data need to demonstrate meaningful therapeutic potential. The benefits of Fast Track designation include opportunities for frequent meetings with the FDA to discuss trial design, development plans and data needed to support drug approval, as well as the ability to submit a registrational filing for approval on a rolling basis, and eligibility for priority review, if relevant criteria are met.

“The FDA’s Fast Track designation reinforces our conviction for the urgent need for new treatments for HCU as well as the promising non-clinical data we have generated in the program to date,” said Aoife Brennan, president and CEO of Synlogic.

Author: Rare Daily Staff