PTC Therapeutics' Huntington's Treatment Gets FDA Fast Track Designation

By Adriano Marchese

PTC Therapeutics' treatment for Huntington's disease has received a fast track designation from U.S. health regulators.

The biopharmaceutical company said Thursday that the Food and Drug Administration granted the designation to the PTC518 program which aims to reduce the production of mutated Huntington protein.

Huntington's disease is a fatal, hereditary, genetic disorder that affects the central nervous system. Due to a defective gene, an abnormal, toxic protein is produced in the body called Huntington protein that damages and kills neurons.

PTC518 was discovered through the development of another drug, Risdiplam, which treats spinal muscular atrophy. In June of this year, PTC518 received positive results from interim data from its Pivot-HD study of the drug in patients with the disease.

With the fast track designation, PTC will be able to develop the treatment quicker and receive a faster review from the FDA.

Write to Adriano Marchese at adriano.marchese@wsj.com

(END) Dow Jones Newswires

September 26, 2024 08:58 ET (12:58 GMT)

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