Friday, September 30, 2022


Biotechnology News Magazine

Amicus Therapeutics Announces Successful Pre-BLA Meeting with U.S. FDA for AT-GAA for the Treatment of Pompe Disease

Latest Posts

City of Hope to Accelerate Immunotherapy Research & Treatment Innovation with $15 Mil Gift from Ted Schwartz Family

Ted Schwartz, who is now cancer free, achieved complete remission at City of Hope in 2020 with the center's leading CAR T cell therapy after a 16-year battle with lymphoma, provided the gift to City of Hope to advance treatment options that offer better outcomes and quality of life for people living with cancer.

Neurocrine Biosciences Appoints Dr Ingrid Delaet as Chief Regulatory Officer

Prior to joining Neurocrine Biosciences, Dr Ingrid Delaet served as Senior Vice President, Regulatory Affairs at Intercept Pharmaceuticals, which she joined in 2016.

Astrea Bioseparations Introduces Nereus LentiHERO, a Fit-for-purpose Solution for Lentiviral Vector Purification

“We believe that AstreAdept will be a game-changer,” explained Astrea Bioseparations’ CEO Terry Pizzie. “Our approach was to rapidly develop and incorporate this material into the Nereus LentiHERO, a simple, fit-for-purpose device that radically transforms how lentivirus can be purified [in terms of speed, recovery, and efficiency].

At Pack Expo, Schreiner MediPharm to Debut Functional Labels Designed from More Sustainable Materials

Schreiner MediPharm advises he new label concepts are based on existing items in Schreiner MediPharm’s roster of functional labeling solutions.

Amicus Therapeutics (NASDAQ:FOLD) today announced the completion of a successful Type B Pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) for AT-GAA (cipaglucosidase alfa co-administered with miglustat), its investigational two-component therapy for the treatment of Pompe disease.

Based on this formal pre-BLA meeting and the final written communication received from the FDA, Amicus intends to complete the rolling BLA submission for cipaglucosidase alfa and submit a New Drug Application for miglustat.

Amicus intends to base its filings on the evaluation of the effects of AT-GAA in late-onset Pompe patients and its safety profile, which will include data from both the Phase 1/2 and Phase 3 PROPEL studies as well as data from the open label extension study. As part of the rolling BLA, Amicus previously submitted the nonclinical component of the cipaglucosidase alfa BLA and is on-track to submit all of the remaining modules of the BLA by the end of the second quarter, including the chemistry, manufacturing and controls (CMC) and clinical sections.

“This meeting with the FDA marks a step forward for thousands of people living with Pompe disease in the United States,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics. “This is an important moment in the development of AT-GAA and a testament to the dedication and perseverance of the patients, physicians and employees who have worked so hard on the development of this investigational medicine. We are moving ahead expeditiously with our submission and all the work necessary to ensure that AT-GAA gets to as many people living with Pompe disease as quickly as possible. “

Jeff Castelli, Ph.D., Chief Development Officer of Amicus Therapeutics, stated, “We believe the data from our clinical trials, including the largest pivotal study ever completed in Pompe disease, have shown clinically meaningful improvements. The AT-GAA development program reflects what we believe is the highest standard in science-based, data-driven, patient-centric therapeutic development. We are confident that we have a robust data package for this regulatory submission, and we look forward to advancing toward its review as soon as possible.”

The U.S. represents the single largest geography for Amicus to positively impact the lives of people with Pompe disease.

AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique enzyme replacement therapy with optimized carbohydrate structures, administered in conjunction with miglustat (AT2221), an orally administered stabilizer of cipaglucosidase alfa.

Previously, the U.S. FDA granted Breakthrough Therapy Designation to AT-GAA for the treatment of late-onset Pompe disease (LOPD) based on clinical efficacy results from the Phase 1/2 clinical study. A rolling BLA for AT-GAA was initiated with the U.S. FDA in the fourth quarter of 2020. Marketing Authorization Applications for AT-GAA are expected to be submitted with the European Medicines Agency (EMA) in the second half of 2021.

AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength.

Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

Latest Posts

Learn More




Our Sister Publication

Medical Device News Magazine