Amicus Therapeutics today announced clinical, regulatory and manufacturing advancements for AT-GAA. AT-GAA is an investigational therapy that consists of two components. First, cipaglucosidase alfa (ATB200), a recombinant human acid alpha-glucosidase (rhGAA) enzyme. Second, miglustat (AT2221), a pharmacological chaperone. The two components together are intended to enhance uptake of the enzyme into the cell.
Amicus is conducting a global Phase 1/2 study (ATB200-02) to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of AT-GAA. This study is ongoing. They have also achieved full enrolment of their Phase 3 PROPEL study (ATB200-03). This is a randomized double-blind study to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa. In their Phase 3 Zip Study, Amicus is enrolling paediatric LOPD patients aged 12-18 years to study pharmacokinetics, safety, efficacy, and pharmacodynamicsof AT-GAA. The primary endpoint of these studies is six-minute walk test (6MWT), while the secondary endpoints includes respiratory function.
The US FDA previously granted Breakthrough Therapy Designation to AT-GAA for the treatment of late onset Pompe disease. Amicus plans to apply for a Rolling Biologics License (BLA) for AT-GAA. The BLA allows Amicus to submit portions of the regulatory application to the FDA as they become available, rather that waiting until it’s all complete.
The British Medicines and Healthcare products Regulatory Agency (MHRA) has issued a Promising Innovative Medicine (PIM) designation for AT-GAA in late onset Pompe disease. This designation means that a clinical trial may be eligible for the Early Access to Medicines Scheme (EAMS), providing earlier access of the treatment to patients.
Process performance qualification (PPQ) runs have been initiated with WuXi Biologics. The PPQ is an important part of the submission for BLA. Manufacturing focus is on building clinical trial and commercial inventory.