FDA of­fers a pro­pos­al to help gener­ic drug de­vel­op­ers get si­mul­ta­ne­ous ap­provals in mul­ti­ple mar­kets

The FDA re­vealed Thurs­day that it’s of­fer­ing a pro­pos­al to the In­ter­na­tion­al Coun­cil on Har­mon­i­sa­tion — ICH — to bet­ter har­mo­nize sci­en­tif­ic and tech­ni­cal stan­dards for gener­ic drugs.

The plan is to let gener­ic drug de­vel­op­ers im­ple­ment a sin­gle glob­al drug de­vel­op­ment pro­gram, with com­mon el­e­ments of ap­pli­ca­tions to file, which will al­low for si­mul­ta­ne­ous ap­provals in mul­ti­ple mar­kets.

“This would make it eas­i­er for de­vel­op­ers that would oth­er­wise on­ly seek gener­ic drug ap­proval in one re­gion to al­so seek ap­proval in the Unit­ed States, in­creas­ing com­pe­ti­tion in Amer­i­ca. And it would al­so make it eas­i­er for de­vel­op­ers that would oth­er­wise on­ly seek gener­ic drug ap­proval in the US mar­ket to gain ac­cess to oth­er mar­kets,” FDA com­mis­sion­er Scott Got­tlieb said in a state­ment.

More specif­i­cal­ly, FDA is propos­ing that ICH de­vel­op a se­ries of guide­lines on stan­dards for demon­strat­ing bioe­quiv­a­lence for both non-com­plex and com­plex dosage forms, and drug prod­ucts. FDA ex­pects that ICH will re­view FDA’s pro­pos­al and that the ICH As­sem­bly will en­dorse the pro­pos­al at its next meet­ing in No­vem­ber 2018.

As far as ar­eas for po­ten­tial con­sid­er­a­tion, There­sa Mullin, as­so­ciate di­rec­tor for strate­gic ini­tia­tives at FDA, told at­ten­dees of the As­so­ci­a­tion for Ac­ces­si­ble Med­i­cines con­fer­ence in Sep­tem­ber, of the pos­si­bil­i­ties:

  • A se­ries of ICH guide­lines on bioe­quiv­a­lence stan­dards for sim­ple and com­plex dosage forms;
  • Asur­vey of ex­ist­ing ICH guide­lines for up­dat­ing as need­ed to in­cor­po­rate rec­om­men­da­tions for gener­ic drugs.

“These ac­tiv­i­ties in­clude pur­su­ing op­por­tu­ni­ties like a har­mo­nized bioe­quiv­a­lence study de­sign that could be ex­pand­ed to in­clude ad­di­tion­al study arms to ac­com­mo­date more than one ref­er­ence prod­uct for bridg­ing pur­pos­es,” Got­tlieb added.

Man­u­fac­tur­ing spec­i­fi­ca­tions may al­so dif­fer be­tween coun­tries.

“For ex­am­ple, right now a spe­cif­ic drug may need to be test­ed un­der dif­fer­ent dis­so­lu­tion meth­ods and ac­cep­tance cri­te­ria to sat­is­fy both the FDA and the Eu­ro­pean Med­i­cines Agency’s reg­u­la­to­ry re­quire­ments,” Got­tlieb said, not­ing that the lack of har­mo­niza­tion across ba­sic com­po­nents of gener­ic drug de­vel­op­ment re­duces the op­por­tu­ni­ties for gener­ic drug de­vel­op­ers to use their da­ta and in­for­ma­tion across mul­ti­ple ap­pli­ca­tions in dif­fer­ent ju­ris­dic­tions.

AAM said in a state­ment that it sup­ports FDA in this ini­tia­tive and looks for­ward to work­ing close­ly with them on it.

FDA al­so re­cent­ly con­duct­ed a pre­lim­i­nary analy­sis that found there are sig­nif­i­cant op­por­tu­ni­ties to ex­pand the avail­abil­i­ty and in­crease mar­ket com­pe­ti­tion for gener­ic drugs be­yond their cur­rent mar­kets.

In par­tic­u­lar, FDA ex­plored the ques­tion of whether there ap­pear to be op­por­tu­ni­ties for coun­tries to gain ac­cess to gener­ic drugs not cur­rent­ly avail­able. In ad­di­tion to the US mar­ket, FDA used da­ta avail­able from 2017 for a sam­pling of nine oth­er coun­tries in­clud­ing five in the EU (France, Ger­many, Greece, Poland and the UK), Japan, Cana­da, Switzer­land and Aus­tralia.

“First, FDA looked at whether the top 100 drugs dis­pensed in the U.S. were al­so avail­able in those nine oth­er coun­tries and found that all of the nine ex­pe­ri­enced some lack of avail­abil­i­ty of these drugs, which ranged from 5 (of 100) un­avail­able in Cana­da to 25 (of 100) un­avail­able in Japan,” Got­tlieb said.

“Next, FDA ex­am­ined the set of ap­prox­i­mate­ly 400 gener­ic drugs with the low­est vol­umes sold in the U.S. used as a proxy for low­est lev­el of U.S. avail­abil­i­ty. FDA found that on­ly 35 per­cent of these drugs were avail­able in the oth­er nine coun­tries and 65 per­cent of these drugs ap­pear to be un­avail­able,” he added.

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.


Zachary Brennan

managing editor, RAPS

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

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A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

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Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

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George Scangos / Credit: Cornell University

ARCH, Soft­Bank-backed Vir Biotech­nol­o­gy un­der­whelms with $143 mil­lion IPO

George Scangos went back to Wall Street, and came back 700 million pennies short.

Scangos’ vaunted startup Vir Biotechnology raised $143 million in an IPO they hoped would earn $150 million. Shares were priced at $20, the low-end of the $20-$22 target.

Launched with backing from ARCH Venture’s Robert Nelsen, Masayoshi Son’s SoftBank Vision Fund, and the Bill & Melinda Gates Foundation, the infectious disease startup was one of a new wave of well-resourced biotechs that emerged with deep enough coffers to pursue a full R&D line rather than slowly build their case by picking off a single lead program.