Alnylam Announces Approval of AMVUTTRA® in Brazil for the Treatment of Transtirretina-Mediated Hereditary Amyloidosis in Adults

Alnylam Pharmaceuticals, a leading RNAi therapy company, has announced that the National Health Surveillance Agency (ANVISA) has approved the registration of AMVUTTRA (vutrisirana sordic) for the treatment of transtirretina-mediated hereditary amyloidosis (hATTR amyloidosis) in adults. HATTR amyloidosis is a progressively disabling and fatal hereditary disease that affects around 5,000 people in Brazil.

“HATTR amyloidosis is considered endemic in Brazil and important unmet needs remain for those patients diagnosed with this rare and complex condition,” explains Dr. Marcia Waddington Cruz, from the Federal University of Rio de Janeiro and researcher of the HELIOS-A study. “The approval of vutrisirana is therefore a very positive novelty for the hATTR amyloidosis community in Brazil, which will now have a new treatment option available. In clinical trials, vutrisirana has demonstrated the potential to disrupt or reverse the progression of the disease, and with its subcutaneous administration once every three months, we expect it to bring additional benefits to patients and their physicians by reducing the burden of treatment.”

“The approval of vutrisirana represents an important milestone for people living with hATTR amyloidosis in Brazil, and our thanks go to the patients, caregivers, physicians and regulatory agents who have made this possible,” said Oliveira Norton, Senior Vice President and Chief operating officer for Latin America at Alnylam. “Vutrisirana is the second RNAi therapy developed by Alnylam to be approved for the treatment of hATTR amyloidosis and we are delighted that patients in Brazil at all stages of the disease will now be able to benefit from this new innovative drug. After approval by ANVISA, the next step is to submit our dossier to the Chamber of Regulation of the Medicines Market (CMED) for price approval and continue to work for the inclusion of vutrisirana in the SUS (Unified Health System).”

Vutrisirana was submitted through the priority review route, granted to innovative medicines for rare diseases. Its approval was based on the results of the HELIOS-A clinical trial, a global, randomized, open-and-center, multicenter phase 3 study that evaluated the efficacy and safety of vutrisirana in a diverse group of patients with hATTR amyloidosis who had polyneuropathy. The results of the HELIOS-A study were published in Amyloid in July 2022. In the HELIOS-A study, vutrisirana reached the primary outcome and all secondary outcomes of the study at 9 months and 18 months, demonstrating reversal of neuropathic impairment and an encouraging safety and tolerability profile.

In June 2022, AMVUTTRA was approved by the U.S. Food and Drug Administration (FDA) for the treatment of hATTR amyloidosis polyneuropathy in adults. In September 2022, AMVUTTRA was approved in Europe by the European Commission (EC) for the treatment of hereditary transtirtin-mediated amyloidosis (hATTR) in adult patients with stage 1 or stage 2 polyneuropathy, and by japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of family-type transtirretina-mediated amyloidosis with polyneuropathy.