FDA lifts clin­i­cal hold on Astel­las’ Pompe gene ther­a­py

The FDA has lift­ed a clin­i­cal hold on Astel­las Phar­ma’s Pompe dis­ease gene ther­a­py tri­al, the Japan­ese com­pa­ny an­nounced Fri­day morn­ing. The FDA put the hold in place last June af­ter in­ves­ti­ga­tors flagged a se­ri­ous case of sen­so­ry nerve dam­age in a pa­tient.

Astel­las says it is mak­ing strides to­ward re­sum­ing dos­ing in the Phase I/II clin­i­cal tri­al for adult pa­tients with late-on­set Pompe dis­ease, in which they lack an en­zyme called GAA that breaks down com­plex sug­ars in the body, caus­ing said sug­ars to build up and dam­age the mus­cles. Astel­las’ head of reg­u­la­to­ry af­fairs David Smethurst de­clined to com­ment on when the tri­al would re­sume dos­ing.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER