Approval of Beyonttra in Brazil brings new treatment options for ATTR-CM
Oral therapy for progressive heart-related disease sold as Attruby in US
Written by |
The oral therapy acoramidis has been approved in Brazil, to be sold as Beyonttra, for treating adults with hereditary or wild-type transthyretin amyloid cardiomyopathy (ATTR-CM).
The regulatory clearance from the Agência Nacional de Vigilância Sanitária “marks an important step forward for Brazilian patients living with ATTR-CM,” Jonathan Fox, MD, PhD, president and chief medical officer of the cardiovascular and renal diseases program at Bridgebio, the therapy’s developer, said in a company press release announcing the agency’s decision.
“[This] is a particularly meaningful advance for patients living in Brazil who have long faced limited options for this progressive, life-threatening disease,” Fox said.
The treatment, marketed under the brand name Attruby in the U.S., is also approved as Beyonttra in the European Union, the U.K., and Japan, with applications under review by regulatory agencies in other regions.
The new approval in Brazil was based mainly on results from a now-complete global Phase 3 clinical trial, called ATTRibute-CM (NCT03860935), that involved more than 600 adults with ATTR-CM. Among participants, Beyonttra treatment led to significant reductions in deaths and cardiovascular-related hospitalizations, along with improvements in physical function and quality of life, meeting all the trial’s main goals.
According to Fábio Fernandes, MD, PhD, an ATTRibute-CM investigator, the trial’s results “represent a major advance for patients with ATTR-CM, who often face an earlier and more aggressive course of disease.”
“For too long, this community has lived with limited options and delayed recognition of their condition,” said Fernandes, who serves as director of the Heart Institute of the Clinical Hospital of the University of São Paulo Medical School in Brazil.
Beyonttra expected to be available in Brazil later this year
Beyonttra will be commercialized in Brazil through a partnership with Biopas, a Swixx Biopharma company that provides innovative medicines to address the unmet medical needs of people living in Latin America. Commercialization efforts are anticipated to start in the second half of this year, according to the company.
“This authorization brings new hope to communities where ATTR-CM is increasingly recognized,” Fox said. “This also reflects our commitment to ensuring that as many patients as possible in as many countries as possible have access to transformative care.”
Fox added: “We are profoundly grateful to the patients, families, investigators, and clinical partners whose courage and partnership made this milestone possible.”
In ATTR-CM, the transthyretin (TTR) protein becomes unstable, breaks apart, and misfolds into toxic clumps known as amyloid fibrils. These clumps, or aggregates, buildup in the heart muscle, causing the heart walls to thicken and stiffen.
This makes it harder for the heart to pump blood effectively, leading to ATTR-CM symptoms such as irregular heartbeat, swelling in the legs and ankles, fatigue, and difficulty exercising.
The disease may be hereditary, caused by mutations in the TTR gene, or wild-type, a form not associated with mutations and that usually develops in older adults.
Available as oral tablets, Beyonttra is a TTR stabilizer, meaning they bind to the TTR protein and help keep it stable. This prevents the protein from breaking apart and forming amyloid deposits. By slowing down amyloid buildup in the heart, the medication is expected to slow disease progression and ease symptoms.
Approval in Brazil, otehr countries, based on positive trial data
The therapy was approved for adults with hereditary or wild-type ATTR-CM in the U.S. in 2024 under the brand name Attruby, and last year, as Beyonttra, in Europe and other regions.
Those approvals were also based on results from ATTRibute-CM, which evaluated the drug’s safety and effectiveness in 632 adults with hereditary or wild-type ATTR-CM enrolled across 18 countries.
Participants were randomly assigned to receive either 800 mg of Beyonttra or a placebo, twice a day for 30 months, or about 2.5 years. Those who completed the trial could enroll in an open-label extension study (NCT04988386), in which all are receiving the medication for up to five years.
ATTRibute-CM results showed that Beyonttra was significantly superior to the placebo across all four measures covered in a main analysis at 30 months. These included death from any cause and rate of cardiovascular-related hospitalizations, as well as changes in blood levels of the heart stress marker NT-proBNP. Changes in the six-minute walk distance (6MWD) test, which measures exercise capacity and physical function, were also assessed.
More recent analyses have demonstrated that Beyonttra reduced the risk of death from any cause by 23% and the risk of cardiovascular-related hospitalizations by half. Benefits in survival and cardiovascular-related hospitalizations were observed as early as three months, the data showed.
Individuals who received Beyonttra were also more likely to see their blood NT-proBNP levels drop than those given the placebo (45% vs. 9%) and walked a mean of 39.6 meters farther during the 6MWD test. Treatment with Beyonttra was also associated with quality of life improvements.
“Seeing a therapy like [Beyonttra] deliver significant reductions in cardiovascular-related hospitalizations, improvements in survival, and preservation of functional capacity and quality of life is profoundly encouraging,” Fernandes said. “These results signal a transformative shift in how we can care for patients across Brazil.”
Another Phase 3 trial, dubbed ACT-EARLY (NCT06563895), is now investigating whether Beyonttra treatment before symptoms appear can prevent of delay disease onset in people carrying disease-causing TTR mutations. That trial is recruiting participants across 24 countries.
Leave a comment
Fill in the required fields to post. Your email address will not be published.