The Agency of Medicines, Food and Medical Devices (ANMAT) in Argentina has approved the use of Galafold as a monotherapy for people ages 16 or older who have alpha-galactosidase A (alpha-GAL A) deficiency due to an amenable genetic mutation.
Amenable mutations now account for 35% to 50% of all Fabry patients worldwide. Galafold’s label in Argentina includes over 350 GLA mutations known to cause the disease and to be sensitive to the treatment.
A list of the mutations that are categorized as “amenable” or “not amenable” to Galafold is available here. Updates to the list with additional GLA variants will be made as these are identified and tested using the Galafold Amenability Assay.
With this regulatory decision, Argentina becomes the first country in Latin America to approve Galafold. Amicus is working with Pint Pharma to complete all the requirements needed to launch the therapy in the upcoming months in that country.
Additional requests for marketing approval of Galafold are under review or planned in other Latin American countries.
“The approval of Galafold in Argentina marks a significant step for our oral precision medicine for Fabry disease in Latin America,” John F. Crowley, chairman and CEO of Amicus Therapeutics, said in a press release. “With more than 400 people known to be living with Fabry disease in Argentina, we look forward to collaborating with Pint Pharma, to bring our patient-dedicated approach to the country.
“As many thousands more are estimated to be living with Fabry in Latin America as a whole, Amicus and our partners are committed to supporting people affected by the disease throughout the region, and we believe a significant portion may have amenable mutations that could benefit from treatment with Galafold,” Crowley added.
Fabry disease occurs due to mutations in the GLA gene, which is responsible for the production of an enzyme called alpha-galactosidase A (AGA). This enzyme works to break down a type of fat called globotriaosylceramide (Gb3 or GL-3) into building blocks that cells can use.
Galafold was designed to restore the activity of the faulty alpha-GAL A enzyme and clear GL3 buildup. It is a chaperone therapy that binds to dysfunctional “amenable mutant” forms of alpha-GAL A, making its structure more stable and partly restoring its activity.
“The approval of Galafold represents a major milestone for patients living with Fabry disease in Argentina and the region,” said David Muñoz, CEO of Pint Pharma. “We are honored to partner with Amicus and are committed to bringing a patient focused vision by supporting early and efficient access to innovative treatments.”
Galafold treatment was seen to improve and stabilize kidney function, reduce heart size, improve the function of the gastrointestinal tract, and significantly reduce blood levels of lyso-Gb3 in patients with classic Fabry disease.
“Galafold has demonstrated meaningful results in previously untreated and treatment-experienced Fabry patients who have amenable mutations,” said Hernan Amartino, MD, head of the Child Neurology Department at Hospital Universitario Austral, Buenos Aires, and an investigator in a migalastat clinical study. “It is exciting for these patients to have a new choice of treatment.”
“We are grateful to those who invest in researching and bringing hope to families that await treatment for so many rare diseases. Even the promise of a new treatment is powerful – it makes us feel less invisible,” said Florencia Braga Menendez, director of patient affairs at Fundación Investigar, an organization working to expand access to healthcare in Argentina. “Today is a big day for Fabry patients and for the advancement of precision medicine in Argentina.”