PTC Announces Tegsedi’s Launch Into Brazilian Market as Treatment for FAP

Ana Pena, PhD avatar

by Ana Pena, PhD |

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Tegsedi (inotersen) has been approved in Brazil to treat stage 1 or 2 nerve damage (polyneuropathy) in adults with hereditary transthyretin amyloidosis (hATTR), also known as familial amyloid polyneuropathy (FAP), PTC Therapeutics announced in a corporate update.

The medicine was granted marketing approval by the Brazilian health regulatory authority. According to PTC Therapeutics, which holds the drug’s commercialization rights in Latin America, Tegsedi’s launch in Brazil is underway, and is being supported by patient and physician services such as medical education and diagnosis efforts. The company anticipates possible revenues for 2020 as a result of this launch.

Approved in the U.S., Canada, and the European Union in 2018, Tegsedi was initially developed by Ionis Pharmaceuticals, with the global marketing licence held by Ionis and its affiliate, Akcea Therapeutics. In 2018, PTC Therapeutics licensed the drug’s commercialization rights for Latin America from Akcea.

The medication is administered via injections under the skin (subcutaneous injections) which can be given in the stomach (abdomen), thigh, or upper arm.

Its active substance is an antisense oligonucleotide, a short piece of lab-made genetic material designed to silence the genetic instructions directing the cell’s production of transthyretin, or TTR, the protein defective in people with FAP.

FAP is caused by mutations in the gene coding for TTR which lead to the accumulation of abnormal clumps of TTR, known as amyloids. These clumps damage tissues and organs around the body, including nerves, and causes the symptoms of the disease.

Tegsedi is designed to interfere with the production of TTR in the liver, the organ responsible for producing most of the protein, thereby preventing the buildup of amyloid deposits in the body and relieving FAP symptoms.

The NEURO-TTR Phase 2/3 trial (NCT01737398) showed that Tegsedi given over 15 months was able to delay nerve cell damage and improve the quality of life of FAP patients, compared with a placebo.

More recent results from an extension study that followed the trial, presented at the 2019 American Academy of Neurology Annual Meeting, show that Tegsedi is able to slow neurological disease progression and improve the well-being of patients over two years of treatment while maintaining the same safety profile.