Date: 2018-09-10
Type of information: Submission of a Market Application in the US
Product name: AT-GAA (ATB200/AT2221)
Compound:
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Action mechanism:
- enzyme replacement therapy. ATB200 is an engineered recombinant human acid alpha-glucosidase (rhGAA) enzyme with an optimized carbohydrate structure, administered in combination with a small molecule pharmacological chaperone (AT2221). In preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced substrate, which was further improved when AT2221 was co-administered with ATB200.
Company: Amicus Therapeutics (USA - NJ)
Disease: Pompe disease
Latest news:
- • On September 10, 2018, Amicus Therapeutics announced regulatory and clinical advancements in its development program AT-GAA for Pompe disease. During the third quarter, Amicus held a Type C meeting with the FDA in order to discuss the regulatory path for AT-GAA. Specifically, Amicus sought input on the design of a pivotal study for full approval for AT-GAA, other supplemental clinical studies in Pompe disease patients, and whether Amicus may pursue an Accelerated Approval pathway in the United States at this time. Amicus has now received final written minutes from the Type C meeting, in which the FDA noted “the importance of expediting new treatments to Pompe patients as fast as possible.” Amicus has incorporated key elements of feedback from the FDA, including the Type C meeting, along with the prior scientific advice received from the European Medicines Agency (EMA) and plans to initiate a pivotal study in 2H18.
- The planned pivotal study, which will compare AT-GAA to the current standard of care, is expected to enroll approximately 100 total Pompe patients. Amicus intends to include both ERT-switch patients and ERT treatment-naïve patients in this single pivotal study to support full approval. The primary endpoint will be 6-minute walk with a primary treatment period of up to 12 months. Patients will be eligible to enroll directly into the pivotal study without participating in the observational study (POM-003). Patients currently enrolled in study POM-003 will be eligible for the pivotal study as well. Amicus also intends to initiate studies in additional patient populations, including pediatric Pompe patients, in 2019.
- With respect to the U.S. regulatory pathway, the FDA also indicated that the current clinical package is not sufficient to support Accelerated Approval. Amicus Therapeutics intends to continue to generate data to support further discussions on a potential pathway for Accelerated Approval with the FDA in 2019, including:
- Data from up to 10 additional ERT-switch patients in a new Cohort 4 as part of the ongoing Phase 1/2 study (data expected in 2019)
- Presentation of longer-term clinical data out to 18-months for the 19 original Phase 1/2 patients (data expected in 2H 2018)
- Completion of a retrospective natural history study in approximately 100 ERT-treated Pompe patients (data expected in 2H 2018)
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
