Amicus Therapeutics is planning a new phase II study of its experimental therapy for Pompe disease, AT2220, now that the FDA has decided to lift a clinical hold on the program. The Cranbury, N.J.-based biotech says it expects to have phase II data available before the end of 2011.
Amicus' drug trials were held after researchers reported that two patients in the initial phase II study experienced serious adverse effects. Researchers were using a high dose as a monotherapy that was likely linked to the side effects.
Researchers now plan to use a combo treatment using an enzyme replacement therapy.