Two gene ther­a­py ap­provals head­line CBER’s FY 2018 re­port

The FDA Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search (CBER) on Wednes­day pub­lished its fis­cal year 2018 re­port, high­light­ing two gene ther­a­py ap­provals, as well as the ap­provals of a he­pati­tis B vac­cine, a vac­cine to pre­vent shin­gles and a flu vac­cine for chil­dren as young as six months.

Both of the gene ther­a­py ap­provals oc­curred at the very be­gin­ning of the pre­vi­ous fis­cal year, with CBER sign­ing off on Spark Ther­a­peu­tics’ Lux­tur­na (voreti­gene nepar­vovec-rzyl) for chil­dren and adult pa­tients with an in­her­it­ed form of vi­sion loss in De­cem­ber 2017 and Gilead’s Yescar­ta (axi­cab­ta­gene ciloleu­cel), a cell-based gene ther­a­py, to treat adult pa­tients with cer­tain types of large B-cell lym­phoma, in Oc­to­ber 2017.

“FY 2018 was par­tic­u­lar­ly ex­cit­ing be­cause of the pace of progress in cel­lu­lar and gene ther­a­pies. CBER re­ceived more than 150 INDs for gene ther­a­py prod­ucts, bring­ing the to­tal num­ber of ac­tive INDs up to near­ly 800 ap­pli­ca­tions,” CBER Di­rec­tor Pe­ter Marks said.

Pe­ter Marks

CBER’s Of­fice of Tis­sues and Ad­vanced Ther­a­pies al­so re­leased 10 new guid­ance doc­u­ments in 2018, in­clud­ing six new draft guid­ance doc­u­ments on gene ther­a­py and a re­cent­ly fi­nal­ized guid­ance on ex­pe­dit­ed pro­grams for re­gen­er­a­tive med­i­cine.

The cen­ter al­so launched a new pro­gram in 2018, known as IN­TER­ACT, where­by spon­sors can seek a pre­lim­i­nary and in­for­mal con­sul­ta­tion with the agency pri­or to a pre-IND meet­ing.

In ad­di­tion, CBER met its drug and med­ical de­vice user fee goals and au­tho­rized the first two donor screen­ing tests for the di­rect de­tec­tion of Zi­ka virus RNA in hu­man plas­ma from in­di­vid­ual donors.

An­oth­er high­light was CBER’s grant­i­ng of an emer­gency use au­tho­riza­tion last Ju­ly to the De­part­ment of De­fense to en­able the emer­gency use of a freeze-dried plas­ma prod­uct man­u­fac­tured by the Cen­tre de Trans­fu­sion San­guine des Ar­mées (French FDP). The prod­uct is used to treat he­m­or­rhage or co­ag­u­lopa­thy of US mil­i­tary per­son­nel in­jured dur­ing com­bat when plas­ma is not avail­able, or its use is not prac­ti­cal. The move fol­lows the Pen­ta­gon’s crit­i­cism of the FDA’s slug­gish­ness in ap­prov­ing the freeze-dried prod­uct, which some said oth­er coun­tries’ troops had used for years.

As part of the FDA’s mu­tu­al recog­ni­tion agree­ments with Eu­rope, CBER as­sessed reg­u­la­tors from 14 coun­tries to en­sure their in­spec­tion of med­ical prod­ucts reg­u­lat­ed by the cen­ter could be rec­og­nized by the FDA. And CBER said it’s con­tin­u­ing to as­sess the re­main­ing EU mem­ber states to meet its 15 Ju­ly com­mit­ment that all mem­ber states are con­firmed.

What the re­port does not men­tion, how­ev­er, is the grow­ing num­ber of Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­py (RMAT) des­ig­na­tion re­quests that the FDA is re­ceiv­ing. Ac­cord­ing to da­ta as of 31 March, there have now been 97 des­ig­na­tion re­quests (in­clud­ing 19 in the first three months of this year), of which 33 have been grant­ed, 53 de­nied and five with­drawn.

CBER FY 2018 Re­port

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

Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

Daniel O'Day [via AP Images]

UP­DAT­ED: Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos — lay­ing out O'­Day's R&D strat­e­gy

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

Altogether, Gilead is gaining rights to 6 clinical-stage assets, 20 preclinical programs and everything else being hatched in translation.

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Hal Barron [File photo]

Hal Bar­ron's team at GSK scores a win with pos­i­tive Ze­ju­la PhI­II front­line study — now comes the hard part

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Alk­er­mes adds bipo­lar I dis­or­der to its FDA wish­list; Con­go con­firms first Ebo­la case in large city

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Pe­ter Kolchin­sky and Raj Shah raise a $300M fund de­vot­ed to biotech star­tups

Peter Kolchinsky and Raj Shah have another $300 million-plus to play with on the biotech venture side of their investment business. 

The two announced Monday morning that they’ve put together their first pure-play venture fund at RA Capital Management, which has been known to bet on just about every angle in healthcare investing — from rounds to follow-on investments at public companies. This new fund of theirs arrives well into a go-go era of new startup financing, with a particular focus on building new biotechs.

Boehringer buys Swiss biotech in its lat­est M&A deal, go­ing the next-gen can­cer vac­cine route

Boehringer Ingelheim has snapped up a Swiss biotech startup and added their group as a new platform for the oncology pipeline. 

The German biopharma company has bagged Geneva-based AMAL Therapeutics, paying out an unspecified upfront in a $358 million deal — cash, milestones and everything else, all in. Plus there’s 100 million euros on the line for commercial milestones.

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Ab­b­Vie beefs up the on­col­o­gy pipeline, bag­ging an up­start STING play­er with its own unique ap­proach

AbbVie isn’t letting its $63 billion buyout of Allergan stop its M&A/deals team from continuing their work.

Monday morning we learned that the pharma giant is snapping up tiny Mavupharma out of Seattle, a Frazier-backed startup that has its own unique take on STING — which is on the threshold of their first clinical trial.

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Billing it­self as the first AI biotech to launch hu­man tri­als, Re­cur­sion adds $121M C round

Billing itself as the first AI biotech with programs in the clinic, Salt Lake City-based Recursion now has a $121 million bankroll to start gathering human data to see if it’s on the right track. 

“We’re trying to build this discovery engine,” Recursion CEO Chris Gibson tells me ahead of the C round news. “We now have the first two programs in the clinic.” And that, he adds, qualifies as a first for any AI establishment “that actually have something in the clinic.”

FDA bats back As­traZeneca's SGLT di­a­betes drug for Type 1 di­a­betes — block­ing a class on safe­ty fears

The FDA has just fired its latest salvo at the SGLT class of diabetes drugs, blowing up some commercial opportunity at AstraZeneca as part of the collateral damage.

The pharma giant reported early Monday that the FDA has rejected its blockbuster drug Farxiga for Type 1 diabetes that can’t be controlled by insulin. And while the pharma giant maintained its usual grim silence in the face of a setback, this one should be easy to interpret.