Sanofi walks away from Alnylam rare disease drug

John Maraganore
Alnylam CEO John Maraganore (Alnylam)

Sanofi has turned down the chance to opt into Alnylam’s primary hyperoxaluria type 1 (PH1) drug lumasiran. The French pharma’s decision gives Alnylam control of a phase 3-ready rare disease drug with an FDA breakthrough therapy designation.

Alnylam granted Sanofi Genzyme the option to pick up rights to its rare genetic disease programs outside of North America and Western Europe in 2014. The provision, and option to take the global rights for one program, survived the recent restructuring of the deal. But Sanofi has opted to stay on the sidelines this time after assessing the phase 1/2 data Alnylam presented late last year.

The look at the ongoing phase 1/2 linked lumasiran to declines in oxalate excretion of more than 50% over baseline. No patients suffered drug-related serious adverse events or dropped out of the trial of the glycolate oxidase-targeted RNAi therapy.


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Alnylam’s results were strong enough for the FDA to award lumasiran breakthrough status but not to get Sanofi to opt in. Sanofi is yet to comment on the thinking behind the decision. But following the January restructuring of the 2014 deal, Alnylam CEO John Maraganore said Sanofi had become more interested in getting global rights to his company’s rare disease drugs.

Applied to Sanofi’s ongoing opt-in option, that thinking suggests the French pharma may turn down the chance to pick up regional rights but exercise its one-time global licensing ticket sometime down the line. Those options expire at the end of next year but could be extended by two years. 

Whatever the thinking, the decision leaves Alnylam in full control of lumasiran as it closes in on phase 3. Alnylam plans to move the drug into a late-phase trial by the end of the year. 

“Based on phase 1/2 clinical data presented to date, we believe lumasiran could be a transformative treatment for patients with PH1, an ultra-rare disease with no approved therapies, in which excess oxalate production leads to kidney failure and significant morbidity and mortality,” Alnylam COO Yvonne Greenstreet said in a statement.

Analysts at Jefferies said in a note: "Though for an ultra-rare disease, lumasiran could be developed by ALNY quickly (in ph.III by YE18), and the Breakthrough Therapy Designation will help de-risk a path to mkt. SNY choosing not to opt-in is not too surprising, given comments they are interested in global rights vs regional and b/c of the ultra-rare indication. The update could have some neg readthrough to DRNA (NC). ALNY success w/ lumasiran could lead to some add'l upside to est."

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