Pres­i­dent Trump names Scott Got­tlieb as the next FDA com­mis­sion­er. What now?

Scott Got­tlieb

Pres­i­dent Trump has named Scott Got­tlieb as the next FDA com­mis­sion­er, re­solv­ing an is­sue that has huge im­pli­ca­tions for the bio­phar­ma in­dus­try and drug de­vel­op­ment.

Got­tlieb’s nom­i­na­tion will be wide­ly ap­plaud­ed by the bio­phar­ma in­dus­try, which will see the move as a com­mit­ment for con­tin­ued re­form with­out the kind of whole­sale dereg­u­la­tion that would scut­tle the agency’s gold stan­dard for drug re­views.

“Thank God it’s Got­tlieb,” mes­saged Baird’s Bri­an Sko­r­ney in his week­ly wrap-up.

As I re­port­ed two weeks ago, Got­tlieb has mapped out an ag­gres­sive re­form agen­da in an­tic­i­pa­tion of this ap­point­ment.

Ac­cord­ing to his state­ments as well as com­ments he’s made to peo­ple fa­mil­iar with his think­ing on the FDA, Got­tlieb in­tends to shoot for the rapid ap­proval of com­plex gener­ics, ush­er­ing in a wave of less ex­pen­sive ri­vals to some of the biggest block­busters on the mar­ket. He’s al­so like­ly to spur the FDA to fol­low the course laid out by agency can­cer czar Richard Paz­dur in speed­ing new ap­provals, pos­si­bly set­ting up a spe­cial unit aimed at or­phan drugs to has­ten OKs with small­er, bet­ter de­signed clin­i­cal tri­als.

Got­tlieb is like­ly to roll back rules re­lat­ed to pre-mar­ket de­vel­op­ment, most no­tice­ably the car­dio­vas­cu­lar out­comes stud­ies for some new meds. A new em­pha­sis on bio­mark­ers as a sur­ro­gate end­point along with an open em­brace of sim­u­la­tion and mod­el­ing could be next.

Oth­er po­ten­tial re­forms in­clude the pos­si­ble quick adop­tion of new de­vices that could be used to  im­prove the kind of medtech Ap­ple, Ver­i­ly and oth­ers have been work­ing on.

Got­tlieb has al­so backed the pub­li­ca­tion of the FDA’s com­plete re­sponse let­ters, de­tail­ing the rea­sons why the agency re­jects a drug. Over the years reg­u­la­tors have rou­tine­ly com­plained that many com­pa­nies have been less than hon­est in re­count­ing the FDA’s po­si­tion on a drug. The FDA is cur­rent­ly re­strict­ed by law in its pub­lic dis­cus­sion about a new drug ap­proval.

The move could spell re­lief for Am­i­cus Ther­a­peu­tics $FOLD and CEO John Crow­ley, who per­son­al­ly lob­bied the pres­i­dent on the FDA’s de­ci­sion to de­lay any fi­nal de­ci­sion on their drug for Fab­ry dis­ease so they could see the re­sults of a safe­ty study in 2019.

Left out in the move: Jim O’Neill. The close as­so­ciate of Pe­ter Thiel, O’Neill fa­mous­ly sug­gest­ed that drugs should be ap­proved based on safe­ty alone, let­ting con­sumers sort out what works. That left many fear­ing that Trump in­tend­ed to toss out the reg­u­la­to­ry frame­work for new drug ap­provals, rais­ing fears that his idea of com­pe­ti­tion would al­low de fac­to place­bos to com­pete for mar­ket share.

Trump has re­peat­ed­ly crit­i­cized bio­phar­ma for out­ra­geous­ly high drug prices, vow­ing to sim­pli­fy the drug de­vel­op­ment process as he al­so sought to dra­mat­i­cal­ly low­er the price of drugs. Got­tlieb will be ex­pect­ed to de­liv­er.

“Among the FDA com­mis­sion­er can­di­dates we’ve read about, Dr. Got­tlieb is well known to in­dus­try and has the req­ui­site ex­pe­ri­ence and tal­ent to re­form and stream­line the FDA ap­proval process,” Hol­land & Knight’s Michael Ga­ba told me re­cent­ly.

One of the rea­sons that Got­tlieb is well known in the in­dus­try is that the physi­cian and for­mer FDA of­fi­cial un­der George W. Bush has plen­ty of di­rect ties to it. As a ven­ture part­ner at New En­ter­prise As­so­ci­ates he’s a board mem­ber at Me­dA­vante, which mar­kets soft­ware for an­a­lyz­ing clin­i­cal tri­al da­ta. And he’s been an ad­vi­sor to Glax­o­SmithK­line, which has shown no ap­petite for rad­i­cal change in the way de­vel­op­ers prove a drug works.

Those ties, along with Trump’s free­wheel­ing com­ments on dereg­u­la­tion, will now be­come tar­gets for De­moc­rats in Wash­ing­ton DC. But un­less there’s a nasty sur­prise hid­den from view, none of it presents a se­ri­ous chal­lenge to his ac­cep­tance as head of the FDA.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

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Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.