FDA gives Rhythm the green light for set­melan­otide, a drug aimed at re­duc­ing obe­si­ty in cer­tain ge­net­ic dis­or­ders

A lit­tle over a year af­ter com­plet­ing suc­cess­ful piv­otal tri­als, Rhythm Phar­ma­ceu­ti­cals $RYTM has its first drug ap­proval on its hands.

The Boston-based biotech an­nounced Fri­day that the FDA gave set­melan­otide the thumbs-up for three rare ge­net­ic dis­or­ders that re­sult in obe­si­ty in pa­tients six and old­er. It’s the agency’s first such ap­proval, Rhythm said, with the in­di­cat­ed de­fi­cien­cies be­ing the POMC, PC­SK1 and LEPR genes. Rhythm will mar­ket the drug as Im­civree, and plans to have it on the shelves in the first quar­ter of 2021.

In­vestors em­braced the news as Rhythm shares shot up more than 20% in Fri­day’s half-day of trad­ing.

Rhythm high­light­ed that ge­net­ic test­ing is re­quired to con­firm the ge­net­ic dis­or­ders and the drug is not in­di­cat­ed for sus­pect­ed or be­nign vari­ant cas­es of POMC, PC­SK1, or LEPR de­fi­cien­cy. It’s al­so not ap­proved for oth­er obe­si­ty-re­lat­ed ge­net­ic dis­or­ders or gen­er­al obe­si­ty.

Set­melan­otide, giv­en by dai­ly in­jec­tion, is an MC4 re­cep­tor ag­o­nist, aim­ing to reg­u­late a path­way that con­trols hunger, en­er­gy ex­pen­di­ture and con­se­quent­ly body weight. The drug is de­signed to re­store im­paired up­stream ac­tiv­i­ty that aris­es from ge­net­ic dis­eases. Pa­tients with the dis­or­der of­ten show signs at a young age, strug­gling with ex­treme hunger that re­sults in obe­si­ty.

“To man­age this obe­si­ty and con­trol dis­rup­tive food-seek­ing be­hav­ior, care­givers of­ten lock cab­i­nets and re­frig­er­a­tors and sig­nif­i­cant­ly lim­it so­cial ac­tiv­i­ties,” said Jen­nifer Miller in a state­ment, a pe­di­atric en­docri­nol­o­gist in­volved with the tri­als. “This FDA ap­proval marks an im­por­tant turn­ing point, pro­vid­ing a much-need­ed ther­a­py and sup­port­ing the use of ge­net­ic test­ing.”

Fri­day’s ap­proval was based on two Phase III tri­als, one study­ing POMC and PC­SK1 de­fi­cien­cies and the oth­er look­ing at LEPR de­fi­cien­cy. In the for­mer, 8 of 10 pa­tients saw greater than 10% weight loss, and the av­er­age re­duc­tion in body weight was 25.4%. For the LEPR study, 5 of 11 pa­tients saw greater than 10% weight loss af­ter a year of treat­ment, with the av­er­age re­duc­tion clock­ing in at 12.5%.

Though those num­bers are small for piv­otal tri­als in gen­er­al, Rhythm said they were the largest con­duct­ed to date in these spe­cif­ic ge­net­ic dis­or­ders. The tri­als were de­signed to in­clude a four-week place­bo with­draw­al pe­ri­od af­ter 12 weeks of treat­ment to eval­u­ate whether pa­tients would re­vert back to their orig­i­nal state. Pa­tients’ on av­er­age gained back 11 pounds dur­ing the with­draw­al pe­ri­od, but once restart­ing on the drug, Rhythm said at the time the trends re­versed.

Im­civree has helped Rhythm chart a steady course since it was found­ed as a pri­vate com­pa­ny back in 2008. The com­pa­ny ul­ti­mate­ly went pub­lic in 2017 af­ter the drug post­ed pos­i­tive Phase II da­ta a year ear­li­er, and now with the ap­proval in hand Rhythm is shoot­ing for the green light in Eu­rope.

The com­pa­ny is cur­rent­ly con­duct­ing an­oth­er Phase III for the drug in Bardet-Biedl or Al­ström syn­drome, with topline da­ta ex­pect­ed in ear­ly 2021 at the lat­est. Rhythm was al­so is­sued a pri­or­i­ty re­view vouch­er and has not stat­ed their plans for it as of yet.

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