Onpattro Approved in Brazil to Treat Adults With FAP

Onpattro Approved in Brazil to Treat Adults With FAP
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Onpattro (patisiran) has been approved in Brazil to treat hereditary transthyretin amyloidosis in adults with stage 1 or stage 2 polyneuropathy, the therapy’s developer, Alnylam Pharmaceuticals, has announced.

Hereditary ATTR amyloidosis, also known as familial amyloid polyneuropathy (FAP), is a genetic disorder that is considered endemic to Brazil, with an estimated prevalence of more than 5,000 cases in the country.

The disease is caused by mutations in the TTR gene, leading to the production of a misfolded transthyretin protein. This misfolded protein forms aggregates (clumps) that accumulate and cause damage to various tissues and organs, resulting in a wide variety of symptoms.

Onpattro is an RNA interference (RNAi)-based therapy that works by lowering the production of misfolded transthyretin, thereby reducing the formation of these harmful aggregates.

The therapy had previously been approved in the U.S., Europe, Japan, Canada, and Switzerland. The new approval by the Brazilian Health Regulatory Agency (ANVISA) marks the first RNAi-based therapy to be authorized for use in Latin America. Onpattro is also the first Alnylam product to be marketed in the region.

“The approval of Onpattro in Brazil marks an exciting milestone for so many Brazilians with [FAP] in need of a new treatment option that could halt the progression of this debilitating and life-threatening disease,” Norton Oliveira, senior vice president, head of Latin America at Alnylam, said in a press release.

“We are grateful to ANVISA for recognizing the significant impact of this disease on patients’ daily lives and for granting approval to the first RNAi therapeutic in Latin America — Onpattro — so swiftly, a mere four months after the marketing authorization application was filed. We will continue collaborating with ANVISA and the Ministry of Health in order to incorporate Onpattro in the federal program (SUS), to make the drug available to patients in need as soon as possible,” he added.

ANVISA’s decision to approve Onpattro was based on data from a Phase 3 trial (NCT01960348) called APOLLO, which assessed the safety and efficacy of the medication, compared to a placebo, in adults with FAP.

From the 225 patients enrolled in APOLLO, 148 people received Onpattro once every three weeks for 18 months, while the remaining 77 participants were given a placebo.

The trial’s findings published in the New England Journal of Medicine showed that Onpattro improved FAP-related symptoms, lowered the levels of abnormal transthyretin in the blood, and improved quality of life, compared to the placebo.

Nearly all participants (97%) experienced adverse events, most of which were mild or moderate in severity. Approximately 20% of the patients who received Onpattro and 10% of those given a placebo had mild or moderate infusion-related reactions. The incidence of severe and serious adverse events was similar in both groups.

ANVISA had previously granted Onpattro priority review, a designation that aims to expedite the review of innovative medications that treat rare diseases.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
Total Posts: 14
Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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