The FDA on Fri­day re­vised the la­bel for Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py Ele­v­idys to add a boxed safe­ty warn­ing and re­strict the ther­a­py’s use to am­bu­la­to­ry pa­tients, re­flect­ing changes that the agency and com­pa­ny pre­vi­ous­ly sug­gest­ed.

The FDA al­so said that it will re­quire Sarep­ta to con­duct an ob­ser­va­tion­al study “to fur­ther as­sess the risk of se­ri­ous liv­er in­jury.” The post­mar­ket­ing study will in­clude around 200 pa­tients — a con­sid­er­able num­ber — and will pe­ri­od­i­cal­ly as­sess their liv­er func­tion for at least a year.

Over the life of the treat­ment, Sarep­ta has on­ly dosed a to­tal of over 1,100 peo­ple with Ele­v­idys across clin­i­cal tri­als and through com­mer­cial chan­nels, the com­pa­ny said in a press re­lease.

Meet the lat­est Chi­nese biotech with meta­bol­ic as­sets ready for part­ner­ing.

Zhe­jiang Do­er Bi­o­log­ics pre­sent­ed mid-stage da­ta that sig­nal that a first-of-its-kind drug is ef­fec­tive at cut­ting high lipid lev­els. The biotech is al­ready in dis­cus­sions with po­ten­tial West­ern col­lab­o­ra­tors.

The prod­uct, named DR10624, is an in­ject­ed tri-ag­o­nist, hit­ting the re­cep­tors for GLP-1, glucagon and FGF21. In a Chi­na-based Phase 2 tri­al in pa­tients with se­vere hy­per­triglyc­eridemia (sHTG), it cut triglyc­eride lev­els by a me­di­an of 69% at the high­est dose, 50 mg per week, af­ter four months.

This was a sta­tis­ti­cal­ly sig­nif­i­cant mar­gin over the 8% re­duc­tion seen with place­bo.

Of the pa­tients giv­en DR10624, 90% achieved triglyc­eride lev­els be­low 500 mg/dL, ver­sus 25% with place­bo. This es­sen­tial­ly means they no longer have sHTG. A re­duc­tion in triglyc­erides of at least 50% was seen in 79% of treat­ed pa­tients, ver­sus 5% giv­en place­bo. Both of these dif­fer­ences were sta­tis­ti­cal­ly sig­nif­i­cant.

The Eu­ro­pean Med­i­cines Agency’s hu­man med­i­cines com­mit­tee (CHMP) rec­om­mend­ed the ap­proval of a Sanofi type 1 di­a­betes drug, Eli Lil­ly’s oral SERD for a cer­tain type of breast can­cer and three oth­er new ther­a­pies.

While the Eu­ro­pean Com­mis­sion has the fi­nal au­thor­i­ty over new drug ap­provals, it typ­i­cal­ly fol­lows CHMP’s rec­om­men­da­tions. Sanofi ac­quired its di­a­betes drug Tzield, or teplizum­ab, in its 2023 buy­out of Proven­tion Bio. The drug was ap­proved in the US in 2022, af­ter show­ing it could de­lay the me­di­an on­set of stage 3 type 1 di­a­betes by about two years.

Stage 3 is when symp­toms usu­al­ly arise for type 1 di­a­betes pa­tients, and it’s al­so when clin­i­cal di­ag­no­sis oc­curs for many pa­tients. Tzield gen­er­at­ed €54 mil­lion in 2024 ($63 mil­lion), up 116% from the year pri­or. In the EU, it will be mar­ket­ed as Teizeild.

The FDA and gener­ic drug­mak­ers kicked off closed-door ne­go­ti­a­tions late last month over the fourth round of user fees, with the agency of­fer­ing up its wish list of changes it wants to see from in­dus­try.

Meet­ing at the FDA's White Oak, MD, cam­pus on Oct. 22, the FDA told in­dus­try rep­re­sen­ta­tives that it wants to help ad­dress the oc­cur­rence of "da­ta fi­deli­ty is­sues," ac­cord­ing to meet­ing min­utes the agency post­ed on Thurs­day. Da­ta in­tegri­ty has been a ma­jor fo­cus for the agency as it says it will look to im­prove trans­paren­cy around man­u­fac­tur­ing fa­cil­i­ty and con­tract re­search or­ga­ni­za­tion (CRO) da­ta.