The U.S. Food and Drug Administration (FDA) has granted a “breakthrough therapy designation” to latozinemab, an experimental treatment for FTD caused by a GRN genetic mutation. The drug is being developed by Alector in partnership with biopharmaceutical firm GSK.

“With this designation, we look forward to continued productive conversations with the FDA, recognizing the unmet need for people living with FTD-GRN, a serious condition for which there are no FDA-approved treatment options available,” said Alector CEO Arnon Rosenthal, PhD in a press release. “Latozinemab, the most advanced progranulin-elevating candidate in clinical development for FTD-GRN, is currently being studied in the pivotal INFRONT-3 Phase 3 study, which achieved target enrollment in October 2023.”

FTD-GRN is caused when inherited variations in the GRN gene impede the ability of the body to produce progranulin, a protein that aids in cell survival and helps regulate inflammation. Lowered levels of progranulin are associated with neurodegeneration.

Latozinemab works by inhibiting a receptor that causes the natural degradation of progranulin. Previous early-stage clinical trials by Alector reported that the drug was “generally safe and well tolerated” by participants of the trial. Progranulin levels were observed to return to normal levels and remained there throughout treatment. By being designated a breakthrough therapy, the drug will benefit from a review process designed to expedite its development.

“With latozinemab now designated a breakthrough therapy, we can expect even greater collaboration between Alector, GSK, and the FDA,” said AFTD Senior Director of Scientific Initiatives Penny Dacks, PhD. “Once the clinical trial concludes, there will be an accelerated decision-making process by regulators on whether to approve the treatment for use in people with FTD-GRN. While latozinemab is a treatment only for people with FTD-GRN, research is already underway investigating treatments for other forms of FTD – we will not stop until there is a treatment for everyone.”

While the Alector trial on latozinemab has stopped enrollment, several other clinical trials are actively seeking people affected by FTD-GRN to test other promising treatments in clinical trials. Visit the Studies Seeking Participants page to learn more about trials that are actively recruiting. Signing up for the FTD Disorders Registry is another way that persons diagnosed, care partners, and family can participate in research.