No­var­tis’ Zol­gens­ma joins grow­ing list of med­i­cines to lose ac­cel­er­at­ed as­sess­ment sta­tus in EU

The EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use (CHMP) re­cent­ly an­nounced its de­ci­sion to re­move No­var­tis’ spinal mus­cu­lar at­ro­phy gene ther­a­py onasemno­gene abepar­vovec from its ac­cel­er­at­ed as­sess­ment pro­gram.

CHMP did not an­nounce its rea­son­ing be­hind the de­ci­sion, which ef­fec­tive­ly means that the treat­ment, ap­proved in the US as Zol­gens­ma and launched at a price of more than $2 mil­lion, will be re­viewed in the EU in 210 days rather than the ac­cel­er­at­ed 150 days. No­var­tis con­firmed that its gene ther­a­py is now un­der stan­dard ap­proval.

A No­var­tis spokesman ex­plained to Fo­cus: “This will give the agency the time they need to re­view the ro­bust amount of da­ta we are pro­vid­ing to an­swer their ques­tions.” An AveX­is (No­var­tis ac­quired AveX­is) spokesper­son al­so told Fo­cus they are work­ing close­ly with Eu­ro­pean reg­u­la­tors and an­tic­i­pate “a po­ten­tial ap­proval in Q4 2019.”

And al­though it’s rare for CHMP to make such a de­ci­sion (on­ly six oth­er med­i­cines have lost their ac­cel­er­at­ed as­sess­ment sta­tus af­ter it was grant­ed in re­cent years), many of these re­ver­sals have come in the last two months.

This month, five med­i­cines, in­clud­ing four can­cer med­i­cines, were tak­en off the ac­cel­er­at­ed as­sess­ment track.

In ad­di­tion to Zol­gens­ma, those in­clud­ed Karyopharm’s mul­ti­ple myelo­ma treat­ment se­linex­or, which was con­tro­ver­sial­ly ap­proved in the US fol­low­ing a neg­a­tive ad­vi­so­ry com­mit­tee de­ci­sion, Stem­line Ther­a­peu­tics’ rare blood dis­ease treat­ment tagrax­o­fusp, which was ap­proved in the US as El­zon­ris last De­cem­ber and Dai­ichi Sankyo’s acute myeloid leukemia drug quizar­tinib, which was re­ject­ed by FDA in June.

Sh­iono­gi’s an­tibac­te­r­i­al ce­fide­ro­col is al­so no longer be­ing re­viewed by CHMP un­der the ac­cel­er­at­ed ap­proval pro­gram, the com­mit­tee said on 9 Ju­ly.

But a de­ci­sion by CHMP to re­move a treat­ment from an ac­cel­er­at­ed as­sess­ment is not al­ways a neg­a­tive sign. For in­stance, Bay­er’s Vi­t­rakvi (larotrec­tinib), which was tak­en out of the ac­cel­er­at­ed as­sess­ment pro­gram in June, then won a rec­om­men­da­tion for con­di­tion­al ap­proval in Ju­ly.

The oth­er two treat­ments to lose their ac­cel­er­at­ed re­views were TaiMed Bi­o­log­ics’ HIV treat­ment ibal­izum­ab, which was ap­proved by the FDA in March 2018 as Trog­a­r­zo, and lost its ac­cel­er­at­ed as­sess­ment sta­tus in the EU in June, and AMMTeK’s neona­tal di­a­betes drug Amg­lidia (gliben­clamide), which was switched at day 90 at the ap­pli­cant’s re­quest to a stan­dard re­view and was lat­er rec­om­mend­ed by CHMP for a mar­ket­ing au­tho­riza­tion in Feb­ru­ary 2018.

As far as why CHMP might de­cide that it is no longer ap­pro­pri­ate to con­duct an ac­cel­er­at­ed as­sess­ment, an EMA guide­line from 2016 points to sev­er­al sce­nar­ios.

“Ex­am­ples of such sit­u­a­tions are when ma­jor ob­jec­tions have been iden­ti­fied that can­not be han­dled in an ac­cel­er­at­ed timetable, when a longer clock-stop is re­quest­ed by the ap­pli­cant (e.g. to pre­pare for the oral ex­pla­na­tion), or when the need for GMP or GCP in­spec­tion be­comes ap­par­ent dur­ing the as­sess­ment,” the guide­line says. “Sim­i­lar­ly, in case of a neg­a­tive trend fol­low­ing the oral ex­pla­na­tion, the CHMP may de­cide to con­tin­ue the as­sess­ment un­der stan­dard as­sess­ment time­lines. The new timetable will be com­mu­ni­cat­ed to the ap­pli­cant and the rea­sons for the change to the stan­dard timetable will be sum­marised in the CHMP as­sess­ment re­port.”

An EMA of­fi­cial’s pre­sen­ta­tion in 2017 al­so shows more than a dozen oth­er med­i­cines from 2013 to 2017 that ob­tained ac­cel­er­at­ed as­sess­ments but lat­er re­vert­ed to stan­dard time­lines.

So­cial im­age: Shut­ter­stock

Ed­i­tor’s note: Up­dat­ed on 7/29 with com­ment from AveX­is and a link to the 2017 EMA pre­sen­ta­tion.

RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

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Alice Shaw, Lung Cancer Foundation of America

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

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