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.

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