Homology Medicines Announces FDA Fast Track Designation for HMI-102 Gene Therapy Development Candidate

Homology Medicines (NASDAQ:FIXX) has announced that the US Food and Drug Administration (FDA) has given it Fast Track designation for HMI-102, a gene therapy to potentially treat adults with phenylketonuria (PKU).

As quoted in the press release:

PKU is an inborn error of metabolism primarily caused by mutations in the PAH gene that lead to phenylalanine hydroxylase (PAH) deficiency resulting in the inability to breakdown phenylalanine (Phe), an essential amino acid found in natural protein.

“We believe FDA’s decision to grant Fast Track designation is a positive step forward for the development of HMI-102, which is designed to treat the underlying cause of this disease and allow freedom from a restrictive diet, the current standard of care,” said Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “The Homology team has been on its own fast track for the past three years working hard to advance its dual gene therapy and gene editing platform from initial concept to the clinic, with the goal­­­­­ of delivering potential cures to patients living with PKU as well as other rare diseases.”

FDA’s Fast Track process is intended to facilitate the development process and expedite review of drugs that are designed to treat serious conditions and fill an unmet medical need to get them to patients earlier. A drug with Fast Track designation can be eligible for more frequent meetings with and written communications from FDA and rolling review of a marketing application, which can lead to earlier drug approval and access by patients.¹

In April 2019, Homology announced that FDA cleared the Investigational New Drug (IND) application for HMI-102 for the treatment of PKU. Previously, the FDA and the European Medicines Agency (EMA) granted orphan drug designation for HMI-102 in the United States and European Union for the use of human hematopoietic stem cell-derived adeno-associated virus AAVHSC15 to treat PAH deficiency, the primary cause of PKU. The Company is currently working with treatment centers to begin the Phase 1/2 pheNIX trial for adult patients with PKU, and expects to report initial clinical data in 2019.

Click here to read the full press release.