Approved as a Long-Term Enzyme Replacement Therapy in Combination with Miglustat for All Adults Living with Late-Onset Pompe Disease
CHMP Opinion for Miglustat, the Oral Enzyme Stabilizer Component of AT-GAA, On-Track for 2Q 2023
PHILADELPHIA, PA, USA I March 27, 2023 I Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the European Commission (EC) has granted approval for Pombiliti™ (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The Company has submitted the previously requested analytical testing for miglustat, the enzyme stabilizer component of AT-GAA. The Committee for Medicinal Products for Human Use (CHMP) opinion for miglustat is expected in the second quarter of 2023.
“Late-onset Pompe disease is a rare, neuromuscular disorder that can have devastating consequences for patients and their families. The European Commission approval for Pombiliti is another major step towards bringing this much needed, new treatment for all adults living in the EU with late-onset Pompe disease. It is the realization of the work of so many individuals and teams dedicated to the mission of improving the lives of people living with Pompe disease,” said John F. Crowley, Executive Chairman of Amicus Therapeutics, Inc.
“We are extremely pleased with the EC approval of Pombiliti, an innovative enzyme replacement therapy that is intended for use in combination with the oral enzyme stabilizer miglustat. We are grateful to the Pompe community who have helped advance this therapy, especially the patients, families, and physicians around the world who participated in our clinical studies. Given the strength of the label and our launch readiness, we believe there is significant commercial opportunity, and that AT-GAA has the potential to become the next standard of care in Pompe disease by redefining the therapeutic expectations of people living with Pompe disease and of their caregivers. Once both components are approved, we look forward to bringing AT-GAA to people in Europe living with LOPD as rapidly as possible,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc.
The EC based its approval on clinical data from the Phase 3 pivotal study (PROPEL), the only randomized, controlled trial in LOPD to include patients in the high unmet need ERT-experienced population, in addition to ERT-naïve patients. The EC approval of Pombiliti follows the positive opinion previously granted by the Committee for Medicinal Products for Human Use (CHMP).
About AT-GAA
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa, a bis-M6P-enriched rhGAA which facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to minimize loss of enzyme activity in the blood. In clinical studies, AT-GAA was associated with demonstrated improvements in both musculoskeletal and respiratory measures.
About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. 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 and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating, including progressive muscle weakness throughout the body, particularly the skeletal muscles and muscles controlling breathing, that worsens over time.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on Twitter and LinkedIn.
SOURCE: Amicus Therapeutics
Post Views: 81
Approved as a Long-Term Enzyme Replacement Therapy in Combination with Miglustat for All Adults Living with Late-Onset Pompe Disease
CHMP Opinion for Miglustat, the Oral Enzyme Stabilizer Component of AT-GAA, On-Track for 2Q 2023
PHILADELPHIA, PA, USA I March 27, 2023 I Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the European Commission (EC) has granted approval for Pombiliti™ (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The Company has submitted the previously requested analytical testing for miglustat, the enzyme stabilizer component of AT-GAA. The Committee for Medicinal Products for Human Use (CHMP) opinion for miglustat is expected in the second quarter of 2023.
“Late-onset Pompe disease is a rare, neuromuscular disorder that can have devastating consequences for patients and their families. The European Commission approval for Pombiliti is another major step towards bringing this much needed, new treatment for all adults living in the EU with late-onset Pompe disease. It is the realization of the work of so many individuals and teams dedicated to the mission of improving the lives of people living with Pompe disease,” said John F. Crowley, Executive Chairman of Amicus Therapeutics, Inc.
“We are extremely pleased with the EC approval of Pombiliti, an innovative enzyme replacement therapy that is intended for use in combination with the oral enzyme stabilizer miglustat. We are grateful to the Pompe community who have helped advance this therapy, especially the patients, families, and physicians around the world who participated in our clinical studies. Given the strength of the label and our launch readiness, we believe there is significant commercial opportunity, and that AT-GAA has the potential to become the next standard of care in Pompe disease by redefining the therapeutic expectations of people living with Pompe disease and of their caregivers. Once both components are approved, we look forward to bringing AT-GAA to people in Europe living with LOPD as rapidly as possible,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc.
The EC based its approval on clinical data from the Phase 3 pivotal study (PROPEL), the only randomized, controlled trial in LOPD to include patients in the high unmet need ERT-experienced population, in addition to ERT-naïve patients. The EC approval of Pombiliti follows the positive opinion previously granted by the Committee for Medicinal Products for Human Use (CHMP).
About AT-GAA
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa, a bis-M6P-enriched rhGAA which facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to minimize loss of enzyme activity in the blood. In clinical studies, AT-GAA was associated with demonstrated improvements in both musculoskeletal and respiratory measures.
About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. 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 and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating, including progressive muscle weakness throughout the body, particularly the skeletal muscles and muscles controlling breathing, that worsens over time.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on Twitter and LinkedIn.
SOURCE: Amicus Therapeutics
Post Views: 81
