EMA validate filing of AT GAA for Pompe disease.
Validation of the application confirms the submission is accepted, and the EMA’s centralized procedure with Committee for Medicinal Products for Human Use (CHMP)’s assessment begins.
The MAAs were submitted to the EMA based on the evaluation of the effects of AT-GAA in adults living with Pompe disease and its safety profile, which include data from the Phase I/II and Phase III PROPEL studies, as well as data from the long-term open-label extension study.
PROPEL study was published in The Lancet Neurology : See- "Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase III trial."- The Lancet Neurology.Vol. 20 No. 12 p1027–1037 Published: December, 2021. Benedikt Schoser,Mark Roberts,Barry J Byrne,Sheela Sitaraman,Hai Jiang,Pascal Laforêt,and others.
Related news and insights
GSK Australia is pleased to announce that the Therapeutic Goods Administration (TGA) has registered Zejula (niraparib) for the treatment of women with advanced high-grade ovarian, fallopian tube or primary peritoneal cancer following completion of first-line platinum-based chemotherapy.
Bristol-Myers Squibb K.K. announced that Japan’s Ministry of Health, Labour and Welfare has approved Abecma (idecabtagene vicleucel), a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with relapsed or refractory (R/R) multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and have either experienced disease progression on the last therapy or relapse after the last therapy.