Akcea Therapeutics and PTC Therapeutics collaborate to commercialize rare disease drugs

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Akcea Therapeutics, Inc. and PTC Therapeutics, Inc. collaborate to commercialize two of Akcea’s rare disease drugs in Latin America Tegsedi and Waylivra. 

“Our decision to partner with PTC to accelerate commercial access for patients in Latin America reflects our commitment to bringing Tegsedi and Waylivra to patients as rapidly as possible,” says Paula Soteropoulos, chief executive officer, Akcea Therapeutics. “PTC’s established rare disease team in Latin America has a proven record of success in patient identification, in physician and patient education and support programs and in efficiently obtaining market access. PTC’s patient-focused approach for rare diseases aligns with ours and will be important as we bring Tegsedi and Waylivra to patients in this region.”

Tegsedi has received marketing authorization approval from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). It is also the subject of a pending new drug application in the U.S. and Canada. Waylivra is under regulatory review in the U.S., Europe and Canada for the treatment of people with familial chylomicronemia syndrome (FCS). It recently received a positive vote from the FDA’s Division of Metabolism and Endocrinology Products Advisory Committee. Waylivra is also in clinical development for Familial Partial Lipodystrophy.

“This collaboration reflects our strategic initiative to leverage PTC’s global infrastructure and reflects our successes in bringing innovative drugs to patients in Latin America,” says Stuart W. Peltz, Ph.D., chief executive officer, PTC Therapeutics. “We are excited to work with Akcea as part of the global launch and commercialization of Tegsedi and Waylivra.”

“One of the key mutations that leads to hATTR amyloidosis occurs more frequently in individuals of Portuguese descent,” says Marcio Souza, chief operating officer, PTC Therapeutics, Inc. “There are approximately 6,000 patients with polyneuropathic hATTR amyloidosis in Latin America, making it a strategically important region for Tegsedi. We are well positioned to file for registration in key Latin American countries in the short term.”


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