image credit: Adobe Stock

FDA lifts hold on Astellas’ Pompe gene therapy

January 20, 2023

The US regulator placed a hold on clinical testing of the gene therapy last June, shortly after a serious case of peripheral sensory neuropathy was reported in the phase 1/2 FORTIS trial in late-onset Pompe disease (LOPD).

That came amid a string of setbacks in Astellas’ gene therapy programmes, including a clinical hold imposed after cases of liver toxicity and deaths were in a trial of its AT132 gene therapy candidate for rare disease X-linked myotubular myopathy (XLMTM), and the halt of three candidates for Duchenne muscular dystrophy (DMD).

Read More on Pharmaphorum