Rep. Frank Pallone (D-NJ) (Francis Chung/Politico via AP Images)

Rep. Frank Pal­lone wants FDA and NIH to act on phar­ma's non­com­pli­ance with tri­al records law

Frank Pal­lone, rank­ing mem­ber of the House En­er­gy & Com­merce Com­mit­tee, crit­i­cized the FDA and NIH to­day for not do­ing enough to pre­vent phar­ma com­pa­nies from fail­ing to re­port cer­tain clin­i­cal tri­al re­sults to the gov­ern­ment data­base, Clin­i­cal­Tri­als.gov.

Point­ing to a re­cent study, Pal­lone said that while com­pli­ance with Clin­i­cal­Tri­als.gov re­port­ing re­quire­ments “has slow­ly but steadi­ly im­proved since 2007, sig­nif­i­cant gaps re­main.”

In a 2021 study of all reg­is­tered tri­als com­plet­ed af­ter Jan­u­ary 2017, spon­sors of on­ly 40% of tri­als re­quired to re­port re­sults did so with­in one year of com­ple­tion, as re­quired by law, and un­der 69% did so at any time.

“FDA, which bears re­spon­si­bil­i­ty for en­forc­ing Clin­i­cal­Tri­als.gov re­quire­ments for a much larg­er num­ber of tri­als, has al­so tak­en very lim­it­ed ac­tion to en­sure com­pli­ance,” Pal­lone wrote. “This is con­cern­ing con­sid­er­ing that it is ap­par­ent when FDA takes ac­tion, it has great ef­fect. FDA first be­gan is­su­ing Pre-No­tice let­ters in 2013, six years af­ter FDAAA was en­act­ed.”

Pal­lone al­so ques­tioned FDA, giv­en the large num­ber of tri­als that ap­pear to be non­com­pli­ant and the suc­cess of FDA’s non­com­pli­ance let­ters so far, whether the agency plans to send these let­ters more fre­quent­ly in the fu­ture. And if not, why not?

The let­ter comes as most NIH-fund­ed clin­i­cal tri­als with re­sults due in 2019 and 2020 did not meet fed­er­al re­port­ing re­quire­ments, the HHS in­spec­tor gen­er­al’s of­fice said in an au­dit re­leased last Au­gust.

“NIH did not have ad­e­quate pro­ce­dures for en­sur­ing that re­spon­si­ble par­ties sub­mit­ted the re­sults of clin­i­cal tri­als, took lim­it­ed en­force­ment ac­tion when there was non­com­pli­ance, and con­tin­ued to fund new re­search of re­spon­si­ble par­ties that had not sub­mit­ted the re­sults of their com­plet­ed clin­i­cal tri­als,” HHS’ OIG re­port says.

Pal­lone lat­er ques­tioned if the process by which FDA re­views these non­com­pli­ant spon­sors should be dif­fer­ent, not­ing:

Is it nec­es­sary to con­duct an in­spec­tion or re­ceive a com­plaint to de­ter­mine whether a spon­sor or tri­al is sub­ject to and in com­pli­ance with Clin­i­cal­Tri­als.gov re­quire­ments, or is FDA able to more ef­fi­cient­ly de­ter­mine this through oth­er means, such as re­view of spon­sor sub­mis­sions or com­mu­ni­ca­tions with spon­sors.

Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

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.

FDA ap­proves GSK's ane­mia drug with safe­ty warn­ing — af­ter bat­ting back sim­i­lar drugs

GSK has secured the first of four US approvals it’s hoping for this year, as the FDA greenlit daprodustat as a treatment for anemia due to chronic kidney disease.

But the FDA limited the use of the drug, to be marketed as Jesduvroq, to patients who have been receiving dialysis for at least four months and stopped short of approving it for patients not dependent on dialysis — in line with the recommendations of the advisory committee it consulted.

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David Kirn, 4D Molecular Therapeutics CEO (via website)

FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

4D Molecular Therapeutics quietly tucked an FDA clinical hold on its Fabry gene therapy into an SEC filing.

Meanwhile, the biotech issued a press release the same day after the closing bell on Thursday touting an IND for another asset, in diabetic macular edema.

The California biotech had paused enrollment of patients in its two trials of the Fabry gene therapy (4D-310) last month after three patients experienced kidney issues, all of which were resolved within four weeks. At the time, 4DMT said it would wait until the second half of this year to look at 12-month clinical data on six patients in the Phase I/II trials, one in the US and one in Taiwan and Australia.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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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.