GAO To Launch In­ves­ti­ga­tion Of FDA’s Or­phan Drug Pro­gram

Act­ing on a re­quest from three in­flu­en­tial U.S. sen­a­tors, the gov­ern­ment’s ac­count­abil­i­ty arm con­firmed that it will in­ves­ti­gate po­ten­tial abus­es of the Or­phan Drug Act.

The Gov­ern­ment Ac­count­abil­i­ty Of­fice still must de­ter­mine the full scope of what it will look in­to and the method­ol­o­gy to be used. De­ter­min­ing the scope will take some months, said Chuck Young, GAO’s man­ag­ing di­rec­tor for pub­lic af­fairs.

Sen­a­tor Or­rin Hatch

Ear­li­er this month, Sens. Or­rin Hatch (R-Utah), Chuck Grass­ley (R-Iowa) and Tom Cot­ton (R-Ark.) sent a let­ter to the GAO and raised the pos­si­bil­i­ty that reg­u­la­to­ry or leg­isla­tive changes might be need­ed “to pre­serve the in­tent of this vi­tal law” that gives drug­mak­ers lu­cra­tive in­cen­tives to de­vel­op drugs for rare dis­eases.

Grass­ley’s of­fice said Tues­day they ex­pect­ed the GAO to be­gin its work in about nine months. The de­lay is typ­i­cal as the agency has a queue of re­quests it is pur­su­ing.

The sen­a­tors have asked the GAO to “in­ves­ti­gate whether the ODA is still in­cen­tiviz­ing prod­uct de­vel­op­ment for dis­eases with few­er than 200,000 af­fect­ed in­di­vid­u­als, as in­tend­ed.”

Con­gress over­whelm­ing­ly passed the 1983 Or­phan Drug Act to mo­ti­vate phar­ma­ceu­ti­cal com­pa­nies to de­vel­op drugs for peo­ple whose rare dis­eases had been ig­nored. Drugs ap­proved as or­phans are grant­ed tax in­cen­tives and sev­en years of ex­clu­sive rights to mar­ket drugs that are need­ed by few­er than 200,000 pa­tients in the U.S.

In re­cent months, re­ports of five- and six-fig­ure an­nu­al price tags for or­phan drugs have am­pli­fied long-sim­mer­ing con­cerns about abuse of the law. The sen­a­tors’ call for a GAO in­ves­ti­ga­tion re­flects that sen­ti­ment.

“While few will ar­gue against the im­por­tance of the de­vel­op­ment of these drugs, sev­er­al re­cent press re­ports sug­gest that some phar­ma­ceu­ti­cal man­u­fac­tur­ers might be tak­ing ad­van­tage of the mul­ti­ple des­ig­na­tion al­lowance in the or­phan drug ap­proval process,” the let­ter states.

In Jan­u­ary, Kaiser Health News pub­lished an in­ves­ti­ga­tion that found the or­phan drug pro­gram is be­ing ma­nip­u­lat­ed by drug­mak­ers to max­i­mize prof­its and to pro­tect niche mar­kets for med­i­cines be­ing tak­en by mil­lions.

That in­ves­ti­ga­tion, which al­so was pub­lished and aired by NPR, found that many drugs that now have or­phan sta­tus aren’t en­tire­ly new. More than 70 were drugs first ap­proved by the Food and Drug Ad­min­is­tra­tion for mass-mar­ket use. Those in­clude cho­les­terol block­buster Crestor, Abil­i­fy for psy­chi­atric dis­or­ders and the rheuma­toid arthri­tis drug Hu­mi­ra, the world’s best-sell­ing drug.

Oth­ers are drugs that have re­ceived mul­ti­ple ex­clu­siv­i­ty pe­ri­ods for two or more rare con­di­tions.

The sen­a­tors asked the GAO for a list of drugs ap­proved or de­nied or­phan sta­tus by the FDA. It al­so asked if re­sources at the FDA, which over­sees the law, have “kept up with the num­ber of re­quests” from drug­mak­ers and whether there is con­sis­ten­cy in the de­part­ment’s re­views.

And they said it would be im­por­tant to in­clude pa­tient ex­pe­ri­ences in the GAO re­view. The GAO does not pro­vide up­dates on on­go­ing work but rather re­ports its find­ings once they com­plete an as­sign­ment.

The rare-dis­ease drugs have be­come in­creas­ing­ly pop­u­lar with phar­ma­ceu­ti­cal and biotech com­pa­nies and are ex­pect­ed to com­prise 21.4 per­cent of world­wide pre­scrip­tion sales by 2022, not in­clud­ing gener­ics, ac­cord­ing to con­sult­ing firm Eval­u­atePhar­ma’s 2017 or­phan drug re­port.

That’s in part be­cause of the ex­or­bi­tant prices that can be charged. Of the top 100 drugs in the U.S., the av­er­age cost per pa­tient per year for an or­phan drug was $140,443 in 2016, com­pared with $27,756 for a non-or­phan, Eval­u­atePhar­ma said.

Orig­i­nal­ly pub­lished by Kaiser Health News. Kaiser Health News, a non­prof­it health news­room whose sto­ries ap­pear in news out­lets na­tion­wide, is an ed­i­to­ri­al­ly in­de­pen­dent part of the Kaiser Fam­i­ly Foun­da­tion.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.