The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Amicus Therapeutics’ lead therapy candidate for the treatment of late-onset Pompe disease, AT-GAA (ATB200/AT221).
The FDA’s decision makes AT-GAA the first investigational therapy for Pompe disease to receive this regulatory status. It also further recognizes the potential of AT-GAA as an attractive strategy to provide symptom relief for people affected by this inherited disorder.
BTD is expected to expedite and support the clinical development and regulatory review of AT-GAA, and will allow Amicus Therapeutics to discuss with the FDA the best strategies to advance the program throughout its development.
“This important Breakthrough Therapy Designation from the FDA reflects the clinical data for our novel Pompe treatment paradigm AT-GAA,” John F. Crowley, chairman and CEO of Amicus Therapeutics, said in a press release. “The BTD here also is based on the significant unmet need that remains for people living with Pompe disease, despite an approved therapy.”
AT-GAA is a combination therapy consisting of a man-made optimized acid alpha-glucosidase (GAA) enzyme — which is faulty in Pompe disease — co-administered with a small molecule that works as a pharmacological chaperone.
Breakthrough status for AT-GAA was supported by clinical evidence resulting from the ongoing Phase 1/2 trial (NCT02675465).
Initial results of the Phase 1/2 trial collected from the first 20 treated patients showed that treatment with AT-GAA could significantly increase the mean levels and activity of GAA enzyme. Also, the stability of the recombinant enzyme increased about 28.6% in the presence of the small molecule chaperone.
AT-GAA significantly improved motor and respiratory function in Pompe patients compared to natural history of the disease, while reducing muscle damage biomarkers.
Still recruiting participants, the trial will explore the efficacy, safety, and tolerability of AT-GAA in comparison to current standard of care (alglucosidase alfa, marketed as Lumizyme and Myozyme) in adults with late-onset Pompe disease over a one-year period.
For more information about the PROPEL study’s locations and contacts, visit the trial page.
“This BTD, together with our results from the Phase 2 study and ongoing PROPEL pivotal study, support our strategy to advance AT-GAA as quickly as possible with the potential to become the new standard of care for all persons living with Pompe disease,” Crowley said.