FDA fi­nal­izes ac­cel­er­at­ed ap­proval la­bel­ing guid­ance

The FDA on Tues­day fi­nal­ized guid­ance on la­bel­ing drugs and bi­o­log­ics ap­proved un­der the ac­cel­er­at­ed ap­proval path­way.

The FDA’s ac­cel­er­at­ed ap­proval pro­gram al­lows the agency to ap­prove prod­ucts to treat se­ri­ous or life-threat­en­ing con­di­tions based on sur­ro­gate or in­ter­me­di­ate clin­i­cal end­points “that are rea­son­ably like­ly to pre­dict clin­i­cal ben­e­fit.” When grant­i­ng ac­cel­er­at­ed ap­proval, FDA will re­quire a spon­sor to com­plete post­mar­ket­ing stud­ies to con­firm the prod­uct’s ben­e­fits.

The nine-page guid­ance fi­nal­izes a draft ver­sion re­leased for com­ment in 2014 and fo­cus­es on the In­di­ca­tions and Us­age sec­tion of la­bel­ing for prod­ucts grant­ed ac­cel­er­at­ed ap­proval based on a sur­ro­gate end­point or a clin­i­cal end­point oth­er than sur­vival or ir­re­versible mor­bid­i­ty.

The guid­ance al­so pro­vides la­bel­ing rec­om­men­da­tions for prod­ucts grant­ed ac­cel­er­at­ed ap­proval that have sub­se­quent­ly had their clin­i­cal ben­e­fit con­firmed, as well as la­bel­ing con­sid­er­a­tions for prod­ucts that have had an in­di­ca­tion with ac­cel­er­at­ed ap­proval with­drawn while oth­er in­di­ca­tions for the same prod­uct re­main ap­proved.

Scott Got­tlieb

“To make sure this path­way re­mains ro­bust, we’re tak­ing new steps to help en­sure that a prod­uct’s la­bel­ing pro­vides ac­tion­able and com­plete in­for­ma­tion about the clin­i­cal ev­i­dence sup­port­ing an ac­cel­er­at­ed ap­proval and clear­ly states that post-mar­ket com­mit­ments may have to be met for an in­di­ca­tion to re­main ap­proved,” said FDA Com­mis­sion­er Scott Got­tlieb.

While the con­tent and for­mat of la­bel­ing for prod­ucts grant­ed ac­cel­er­at­ed ap­proval “is in most ways the same as la­bel­ing for drugs with tra­di­tion­al ap­proval,” prod­ucts grant­ed ac­cel­er­at­ed ap­proval based on a sur­ro­gate or in­ter­me­di­ate clin­i­cal end­point are re­quired to in­clude a “suc­cinct de­scrip­tion of the lim­i­ta­tions of use­ful­ness of the drug and any un­cer­tain­ty about an­tic­i­pat­ed clin­i­cal ben­e­fits,” the guid­ance says.

The FDA al­so says that the In­di­ca­tions and Us­age sec­tion should ac­knowl­edge that prod­uct was grant­ed ac­cel­er­at­ed ap­proval that may be con­tin­gent up­on ver­i­fi­ca­tion of clin­i­cal ben­e­fit.

In some cas­es, FDA says that sim­ply de­tail­ing the end­point that was used to sup­port ac­cel­er­at­ed ap­proval is suf­fi­cient to con­vey the lim­i­ta­tions of use­ful­ness and clin­i­cal ben­e­fit un­cer­tain­ty. How­ev­er, the agency says that in oth­er cas­es “ad­di­tion­al con­text about the ap­proval should be in­clud­ed by iden­ti­fy­ing the clin­i­cal out­come(s) that are ex­pect­ed … but not yet es­tab­lished.”

Spon­sors are al­so ad­vised to in­clude a brief sum­ma­ry of any re­quired post­mar­ket­ing stud­ies to “fur­ther em­pha­size the lim­i­ta­tion of the clin­i­cal study re­sults sup­port­ing the ac­cel­er­at­ed ap­proval.”

Af­ter clin­i­cal ben­e­fit has been ver­i­fied, spon­sors are in­struct­ed to re­vise the in­for­ma­tion in the In­di­ca­tions and Us­age sec­tion to “re­flect the pop­u­la­tion and con­di­tion for which there is sub­stan­tial ev­i­dence of ef­fec­tive­ness, in­clud­ing any new or re­main­ing lim­i­ta­tions of use.”

Ad­di­tion­al­ly, the FDA says that spon­sors should re­vise oth­er sec­tions of the la­bel­ing to re­flect da­ta from the con­fir­ma­to­ry stud­ies, such as the Ad­verse Re­ac­tions and Clin­i­cal Stud­ies sec­tions.

For prod­ucts whose ac­cel­er­at­ed ap­proval is with­drawn that re­main ap­proved for oth­er in­di­ca­tions, FDA says that spon­sors must up­date the la­bel­ing to re­move in­for­ma­tion re­lat­ed to the with­drawn in­di­ca­tion.

How­ev­er, in cer­tain cir­cum­stances, spon­sors may be re­quired to add new in­for­ma­tion to the la­bel­ing con­cern­ing a with­drawn in­di­ca­tion, such as when an in­di­ca­tion is with­drawn for lack of ev­i­dence or when an in­di­ca­tion is with­drawn for safe­ty is­sues.


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.

Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Partners Innovation Fund

David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.

Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.