The two drugs are RNAi treatments. This means they are designed to prevent RNA interference — a DNA sequence’s ability to turn off the gene that the sequence is part of. The silencing leads to the production of the faulty transthyretin protein that causes FAP.
Alnylam now has the global rights to both treatments, and will fund their development and commercialization on its own.
In return for giving up its share of the rights to patisiran and ALN-TTRsc02, Sanofi will receive royalties when the products reach the market. Sanofi will also obtain rights to the hemophilia treatments that the companies were developing under another partnership.
“This strategic restructuring enables streamlined development and an optimized approach to bringing innovative medicines to patients with ATTR amyloidosis and hemophilia around the world,” said Dr. John Maraganore, Alnylam’s chief executive officer. ATTR amyloidosis is another name for familial amyloid polyneuropathy.
“This will allow us to develop both products [patisiran and ALN-TTRsc02] in a comprehensive manner, potentially addressing the full spectrum of transthyretin-mediated amyloidosis disease treatment and prevention,” he added. Still another name for familial amyloid polyneuropathy is transthyretin-mediated amyloidosis.
Alnylam has asked both U.S. and European regulators to approve patisiran as an FAP therapy. Believing it to be a significant improvement in the treatment of the disease, both are giving Alnylam’s application accelerated review.
Patient advocacy organizations are also supporting patisiran.
The head of the Amyloidosis Research Consortium hailed Alnylam’s application for FDA approval of the treatment as a milestone.
“We are hopeful this therapy will be approved for patients in the U.S. who are desperately awaiting treatment options,” Isabelle Lousada, the organization’s president and chief executive officer, said in a press release at the time.
“In the year ahead, we look forward to the potential global commercial launch of patisiran, the first-ever RNAi therapeutic to enter the market,” Maraganore said in a 2018 corporate goal announcement. Its approval would mark “the birth of a whole new class of medicines with the potential to transform the treatment of diseases with high unmet need,” he said.
Meanwhile, the company plans a Phase 3 clinical trial of its other RNAi treatment for FAP — ALN-TTRsc02 — by the end of 2018.
Like patisiran, ALN-TTRsc02 is designed to prevent the production of the transthyretin protein that is faulty in FAP. It does this by binding to and destroying messenger RNA, an intermediary molecule in the production of protein.