FDA health ad­vis­ers back first-in-class choles­terol drug


Fed­eral health ad­vis­ers on Tues­day rec­om­mended ap­proval for a highly an­tic­i­pated choles­terol drug from Sanofi and Re­gen­eron Phar­ma­ceu­ti­cals, but with the caveat that more data is needed about its long-term abil­ity to re­duce heart at­tacks.

The ex­pert panel rec­om­mended by a 13-3 vote that the Food and Drug Ad­min­is­tra­tion ap­prove the in­jectable drug, called Pralu­ent.

But in an un­ex­pected devel­op­ment, a num­ber of pan­elists said the drug should only be used in pa­tients with ab­nor­mally high choles­terol lev­els caused by an in­her­ited dis­or­der. Those pan­elists said they wanted to see more data about whether the drug ul­ti­mately re­duces heart prob­lems, be­fore it is used more broadly.

If the FDA fol­lows the group’s rec­om­men­da­tion, the drug could be ap­proved for a much smaller pop­u­la­tion than its de­vel­op­ers have pro­posed.

Pralu­ent is part of a new class of in­jectable drugs that lower choles­terol more than older med­i­ca­tions called statins, which have been the stan­dard treat­ment for more than 20 years. A sim­i­lar drug from Am­gen will go be­fore the same panel on Wed­nes­day.

Both drugs block a sub­stance called PCSK9, which in­ter­feres with the liver’s abil­ity to re­move choles­terol from the blood. An­a­lysts ex­pect the drugs to grow into block­buster sell­ers, gen­er­at­ing bil­lions of dol­lars in new sales. But Tues­day’s meet­ing un­der­scores the un­cer­tainty about who should re­ceive such med­i­ca­tions.

More than 73 mil­lion U. S. adults, or nearly one-third, have high LDL choles­terol, ac­cord­ing to the Cen­ters for Dis­ease Con­trol and Pre­ven­tion. Those pa­tients have twice the risk of heart dis­ease, the lead­ing cause of death in the U.S.

Paris-based Sanofi and Re­gen­eron, which is based in Tar­ry­town, New York, have sug­gested mar­ket­ing their drug to a large group of pa­tients, in­clud­ing those who are at high-risk for heart at­tacks and those who can­not tol­er­ate statin drugs due to side ef­fects.

But many pan­elists said they would limit the drug’s use to a much nar­rower group of pa­tients who have ab­nor­mally high choles­terol lev­els caused by a fam­ily dis­or­der.

Key to the panel’s de­ci­sion was whether low­er­ing bad, or LDL, choles­terol trans­lates into re­duced heart at­tacks. For over 20 years, the FDA has used re­duced LDL lev­els as a bench­mark to ap­prove choles­terol drugs, in­clud­ing statins like Lipi- tor and Zo­cor.

But re­cent stud­ies of drugs like niacin have shown that re­duc­ing choles­terol lev­els does not al­ways lead to re­duc­tions in heart attack, stroke and re­lated prob­lems.

The FDA’s in­ter­nal sci­en­tists pressed pan­elists on whether they thought lower LDL choles­terol was still an ac­cu­rate pre­dic­tor of a drug’s ben­e­fit.

De­spite the mea­sured en­dorse­ment, com­pany rep­re­sen­ta­tives said they were “grat­i­fied” by the meet­ing’s out­come.

Re­gen­eron’s stock fell about 3 per­cent in ex­tended trad­ing fol­low­ing the an­nounce­ment. Sanofi’s U.S.-traded shares rose about 1 per­cent.

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