EMA, Regulatory

EMA committee recommends Alnylam’s early-stage ATTR amyloidosis drug for orphan status

With the spotlight on patisiran’s likely imminent approval, Alnylam $ALNY wants to show some love for its follow-on drug for transthyretin-mediated (ATTR) amyloidosis, which is close to becoming an orphan drug in Europe.

The EMA’s Committee for Orphan Medicinal Products issued a positive opinion recommending ALN-TTRsc02, with an official designation by the European Commission expected to follow. Like patisiran, the drug is designed to halt production of TTR protein before it’s made.

Boasting its “second generation” chemistry, low volume and sparse dosing schedule, Alnylam is looking to advance the product into Phase III later this year.

Rena Denoncourt

“With potent and durable TTR knockdown, we believe ALN-TTRsc02 holds great promise as an investigational, once-quarterly, low volume, subcutaneously administered RNAi therapeutic for the treatment of a broad spectrum of patients with ATTR amyloidosis,” said ALN-TTRsc02 program leader Rena Denoncourt in a statement.

The news comes after Alnylam restructured its deal with Sanofi to gain broader control over its ATTR amyloidosis portfolio, handing global rights to a hemophilia drug to its partner in return.

“This will allow us to develop both products in a comprehensive manner, potentially addressing the full spectrum of transthyretin-mediated amyloidosis disease treatment and prevention,” CEO John Maraganore said at the time.


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Director/Sr Director, Oncology/Immuno-Oncology
Aro Biotherapeutics Company Denver, CO
Director/Sr. Director Oncology
Aro BioTx Philadelphia, PA
Manufacturing Associate – Upstream
Molecular Templates Austin, TX
Scientist, Process Development
Molecular Templates Austin, TX

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