PTC Therapeutics to commercialise Akcea’s two rare disease drugs

Ionis Pharmaceuticals affiliate Akcea Therapeutics has signed an agreement with biopharmaceutical company PTC Therapeutics for the commercialisation of its rare disease drugs Tegsedi (inotersen) and Waylivra (volanesorsen) in Latin America.

Under the terms of the agreement, PTC will obtain exclusive rights for the medications in Latin America and some Caribbean markets.

Tegsedi is an antisense oligonucleotide (ASO) inhibitor targeting production of human transthyretin (TTR). It has gained the European Commission (EC) marketing authorisation to treat stage 1 or 2 polyneuropathy in adults suffering from hereditary transthyretin amyloidosis (hATTR amyloidosis).

Additional regulatory submissions for the drug have been made in the US and Canada.

Waylivra is currently under review in the US, Europe and Canada for treating familial chylomicronemia syndrome (FCS). The drug is also being developed to treat familial partial lipodystrophy (FPL) patients.

Akcea Therapeutics CEO Paula Soteropoulos said: "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.

"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."

As part of the agreement, PTC Therapeutics will pay $18m as an upfront licensing fee to Akcea. Of the total fee, $12m is to be paid on signing and $6m will be paid on Waylivra’s approval in the US or European Union (EU), whichever is earlier.

PTC will pay an additional $8m in regulatory milestones along with royalties on net sales of each drug in its territory.

Akcea obtained the global rights to the rare disease drugs through licences from Ionis. Ionis will gain 60% of the milestone payments and royalties paid by PTC for Tegsedi, while the remaining 40% will be retained by Akcea.

In the case of Waylivra, Akcea and Ionis will equally split the milestone payments and royalties.

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