Sanofi subsidiary Genzyme and Alnylam Pharmaceuticals have expanded their strategic agreement to develop and commercialise new treatments for rare genetic diseases.

As part of the deal, Genzyme will have rights to Alnylam’s portfolio of clinical and pre-clinical stage drug candidates, while Alnylam will retain most product rights in North America and Western Europe.

Alnylam will also have significantly expanded development and commercial opportunities for its genetic medicine pipeline via Genzyme’s global infrastructure in rare diseases.

Genzyme president and CEO David Meeker said: "It strengthens our pipeline and provides us with the opportunity to meet the needs of patients with rare diseases around the world through our well-established global organisation.

"This transaction also powerfully underscores Sanofi’s commitment to investing in Genzyme as one of the company’s key growth drivers. Our partnership with Alnylam has been highly collaborative, and their world-class RNAi technology holds the promise to provide a platform for sustained drug development for rare genetic diseases for years to come."

In 2012, both firms formed an exclusive alliance to develop and commercialise Alnylam’s lead product, patisiran, which is currently in Phase III development and intended for the treatment of transthyretin (TTR)familial amyloid polyneuropathy, a rare life-threatening disease that damages the nervous system.

Under the expanded deal, Genzyme will obtain expanded rights to patisiran and also obtain rights to globally commercialise three products in Alnylam’s pipeline.

Under the terms of the agreement, Genzyme and Alnylam will co-develop and co-commercialise ALN-TTRsc, a product currently in Phase II development for the treatment of familial amyloid cardiomyopathy, in North America and Western Europe, while Genzyme markets the product in the rest of world.

In addition, Genzyme will have the rights to two additional products after completion of early clinical trials and will be able to choose between full global rights or co-marketing rights, depending on the product.

"It strengthens our pipeline and provides us with the opportunity to meet the needs of patients with rare diseases around the world."

The company will also have the option up until 2020, with the possibility of extension through the end of 2021, to develop and commercialise outside of North America and Western Europe all products being developed to treat rare genetic diseases from Alnylam’s pipeline.

Genzyme will also become a major Alnylam shareholder through an upfront purchase of $700m of newly issued stock at approximately $80/share, representing about 12% ownership position.

Pursuant to the terms of the expanded pact, Alnylam will receive R&D funding, starting on 1 January 2015, for programs where Genzyme has elected to opt-in for development and commercialisation. Furthermore, Alnylam is eligible to receive milestones and royalties.

Alnylam chief executive officer John Maraganore said: "It is a game-changer for both the advancement of RNAi therapeutics as a new class of genetic medicines to patients around the world, and for our commitment to build a leading, independent biopharmaceutical company that delivers value to our shareholders.

"In this new alliance, Alnylam benefits enormously from Genzyme’s proven global capabilities, enabling us to accelerate and expand market access for our ‘Alnylam 5×15’ products."

Image: Genzyme global headquarters in Cambridge, Massachusetts, US. Photo: courtesy of Rotatebot.