Once re­ject­ed, FDA now rolls out a short red car­pet for Am­i­cus’ mi­gala­s­tat

John Crow­ley

Am­i­cus CEO $FOLD John Crow­ley must have made a very fa­vor­able im­pres­sion on the FDA as he lob­bied se­nior of­fi­cials to over­turn their re­jec­tion of his ex­per­i­men­tal Fab­ry drug mi­gala­s­tat.

Not on­ly did reg­u­la­tors just ac­cept the fil­ing for a new re­view, the agency is giv­ing it pri­or­i­ty sta­tus, cut­ting the re­think to 6 months and flag­ging a clear in­di­ca­tion that it’s an odds-on fa­vorite for an of­fi­cial mar­ket­ing OK .

Cut­ting 4 months off the re­view process, the FDA will have its new de­ci­sion out by Au­gust 13.

An­a­lysts are al­ready count­ing the near-term rev­enue that Am­i­cus stands to earn. Joseph Schwartz at Leerink not­ed:

With the PDU­FA goal date of Aug 13, 2018, US rev­enue could con­tribute to FY2018 rev­enue guid­ance of $75M-$85M an­nounced last month. We con­tin­ue to see strong growth dri­vers for Am­i­cus this yr. as the com­pa­ny con­tin­ues to ad­vance its mi­gala­s­tat (Galafold; Fab­ry dis­ease) and ATB200/AT2221 (Pompe dis­ease) pro­grams through the clin­ic.

Crow­ley had em­ployed some du­bi­ous po­si­tions as he fought against the re­jec­tion, which came with a de­mand from the FDA for a new Phase III study of gas­troin­testi­nal symp­toms. Ini­tial­ly, the CEO had told in­vestors it would take 2 years to come up with the da­ta. But he told Janet Wood­cock and oth­ers that the ac­tu­al time­line would be 5 to 7 years, which made the de­vel­op­ment pro­gram im­prac­ti­cal.

Scott Got­tlieb

This was one of three drug pro­grams that the FDA re­versed it­self on in the months af­ter Scott Got­tlieb took over as com­mis­sion­er at the FDA, with Eli Lil­ly and Ther­a­peu­tic­sMD — chaired by ex-HHS sec­re­tary Tom­my Thomp­son — get­ting a do-over. The agency has nev­er ex­plained the re­jec­tions, nev­er ex­plained why it de­cid­ed to re­con­sid­er their de­ci­sion, or why it would of­fer Am­i­cus a quick de­ci­sion now.

But they nev­er do, re­quired by the rules to stay qui­et. Got­tlieb had ini­tial­ly said he would press for the redact­ed pub­li­ca­tion of com­plete re­sponse let­ters like these, but he re­cent­ly shrugged that off.

“Con­grat­u­la­tions, again!” Crow­ley wrote in a note to Got­tlieb at­tached to his case for a re­view. “As dis­cussed, the hopes and well be­ing of so many liv­ing with rare, dev­as­tat­ing dis­eases rests now with your great lead­er­ship and wis­dom. Thanks for your at­ten­tion to the at­tached.”

Got­tlieb’s staff told me that the com­mis­sion­er nev­er saw that pack­et, redi­rect­ing it to oth­er se­nior of­fi­cials at the FDA. Of­fi­cials, though, de­clined to ex­plain what Crow­ley meant by the word “again,” or if Got­tlieb had dis­cussed the is­sue with the CEO.

That note, along with Crow­ley’s as­ser­tions on the time­line to re­spond, were ob­tained un­der FOIA by the Pro­ject on Gov­ern­ment Over­sight.

Got­tlieb has open­ly ad­vo­cat­ed for a more ef­fi­cient re­view process, par­tic­u­lar­ly where rare dis­ease are con­cerned. That al­so squares with Pres­i­dent Don­ald Trump’s vow to speed up the reg­u­la­to­ry process. And af­ter start­ing out with a high rat­ing from the in­dus­try, biotech ex­ecs are now cheer­ing him on as one of the best things that’s hap­pened at the FDA in years.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Teresa Graham, incoming Roche Pharmaceuticals CEO

In­com­ing Roche CEO builds out his top team, tap­ping Genen­tech vet to lead phar­ma di­vi­sion

Roche announced another leadership shuffle Thursday morning – the head of global product strategy, Teresa Graham, will take over as CEO of Roche Pharmaceuticals in March while the company’s corporate executive committee will make a spot for Levi Garraway, CMO and executive VP of global product development.

Thomas Schinecker will take over the top spot as Roche group CEO in March, leaving his spot as head of diagnostics.

Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.

BeiGene's new website helps direct cancer patients and caregivers to a wide variety of sources for help.

BeiGene re­veals men­tal health and can­cer care gap in study, de­buts dig­i­tal re­sources

One-fourth of cancer patients are living with depression — and another 20% suffer from anxiety. That’s according to new study results from BeiGene, conducted by Cancer Support Community (CSC), about the mental and emotional health of cancer patients.

While the fact that people with cancer are also dealing with depression or anxiety may not be surprising, what is — and was to BeiGene — is that a majority of them aren’t getting support. 60% of respondents said they were not referred to a mental health professional, and even more concerning, two in five who specifically asked for mental health help did not get it. CSC, a nonprofit mental health in cancer advocacy group, surveyed more than 600 US cancer patients.

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One of the paintings from Gilead's latest campaign making AI art to help MBC patients be 'seen and heard.'

Gilead com­bines ar­ti­fi­cial in­tel­li­gence and art to draw at­ten­tion and hope to MBC

What if you could “see” the emotions and feelings of people living with metastatic breast cancer? That’s what Gilead Sciences’ agency VMLY&R Health did last year, using artificial intelligence and sound analytics to turn the interviews of three women living with metastatic triple-negative breast cancer into works of art.

Using the sound waves, a robotic painting device translated their stories of struggle and hope into colors, contours and brush strokes. The result? An art exhibition called “Paintings of Hope” that was first displayed at ESMO in September in Paris, but has since traveled to hospitals and medical conferences in Europe and Spain.

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Giovanni Caforio, Bristol Myers Squibb CEO (Nicolas Messyasz/Sipa via AP Images)

Bris­tol My­ers turns at­ten­tion to new prod­ucts in wake of Revlim­id patent loss

Bristol Myers Squibb CEO Giovanni Caforio is shifting his focus to newer products as generic sales continue to gnaw at the company’s blockbuster myeloma drug Revlimid.

Both Revlimid and Abraxane sales took a dive last year thanks to generic rivals, BMS reported in its Q4 and full-year results on Thursday. As a result, Q4 sales dipped 5% and full-year sales remained flat. However, Caforio sees a silver lining — or rather, two of them.

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