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FDA lifts hold on Astellas gene therapy for Pompe disease

Dive Brief:

  • The Food and Drug Administration has cleared Astellas Pharma to resume testing of its gene therapy for the rare and inherited metabolic disorder known as Pompe disease.


  • The regulator stopped an early-stage study of the treatment in June over safety concerns after a patient developed mild nerve damage. That hold has been lifted, Astellas said in a statement Friday, and the company is now working to complete “clinical and regulatory activities necessary to resume dosing” in the trial.


  • The FDA’s decision gives something of a boost to Astellas, which has faced repeated safety setbacks in its gene-based programs since it acquired them in 2019 with its $3 billion purchase of Audentes Therapeutics.

Dive Insight:

Like its peers, Astellas has sought to capitalize on advances in gene therapy research. But it has faced significant delays and setbacks that have forced it to trim its pipeline of gene-based programs.

In April, the Japanese drugmaker scrapped three experimental treatments for Duchenne muscular dystrophy. The decision contributed to $700 million in impairment charges for its gene therapy assets.

The company has also faced repeated holds on its testing of a treatment called AT132 for a rare neuromuscular disease known as X-linked myotubular myopathy. The study remains on hold after four children died from liver damage and resulting fatal complications.

The FDA’s hold on the Pompe disease trial came after a patient developed peripheral sensory neuropathy, a type of nerve damage that can include symptoms like numbness or pain in the hands and feet. The investigator graded the side effects as mild in severity but judged it to be serious due its “medical significance,” Astellas said at the time.

The treatment, called AT845, is meant as a one-time treatment for Pompe, which is typically treated with medications that replace the protein that’s missing in people with the condition.

“We look forward to resuming the FORTIS clinical trial and the continued development of AT845 as an important potential new treatment for adults living with [late-onset Pompe disease],” Astellas’ medical director Ha Tran said in a statement.

Others in the Pompe space have also faced delays.

The FDA in October delayed its review of Amicus Therapeutics’ treatment for Pompe because it has been unable to inspect the company’s facilities in China. Amicus expects the FDA to still approve its therapy, called AT-GAA, which is a recombinant version of the missing protein modified to improve its uptake in the body.