FDA's can­cer drug ad­vi­sors will re­view 2 more dan­gling ac­cel­er­at­ed ap­provals for mul­ti­ple myelo­ma, leukemia

In an at­tempt to get its house of ac­cel­er­at­ed ap­provals in or­der, the FDA is hold­ing its sec­ond ad­comm of 2021 to re­view can­cer drugs that won ac­cel­er­at­ed ap­provals but failed to con­firm clin­i­cal ben­e­fit in sub­se­quent tri­als or have tak­en a long time to read those da­ta out.

On Dec. 2, the FDA’s On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee will re­view two ac­cel­er­at­ed ap­provals from Se­cu­ra Bio’s Fary­dak (panobi­no­s­tat), a third-line mul­ti­ple myelo­ma drug, and Acrotech Bio­phar­ma’s Mar­qi­bo, as a third-line drug for adult pa­tients with Philadel­phia chro­mo­some neg­a­tive acute lym­phoblas­tic leukemia. Both drugs have been mar­ket­ed for more than five years un­der their ac­cel­er­at­ed ap­provals but have record­ed neg­li­gi­ble sales in their re­spec­tive in­di­ca­tions in re­cent years.

More in­for­ma­tion on what ex­act­ly hap­pened with both drugs’ con­fir­ma­to­ry tri­als will be­come clear­er two days ahead of the meet­ing, when FDA re­leas­es its brief­ing doc­u­ments. The com­mit­tee will be tasked with de­cid­ing whether these in­di­ca­tions should re­main on the mar­ket while ad­di­tion­al tri­al(s) are con­duct­ed, the agency said in Thurs­day’s Fed­er­al Reg­is­ter.

In the case of Fary­dak, No­var­tis won the ini­tial ac­cel­er­at­ed ap­proval in 2015 but said it like­ly wouldn’t start the con­fir­ma­to­ry tri­al for al­most 3 years, and the FDA gave the com­pa­ny un­til this year to fin­ish both of its re­quire­ments. In March 2019, Se­cu­ra Bio then bought the drug from No­var­tis, but sales have fall­en be­low $100 mil­lion in re­cent years, and the ac­cel­er­at­ed ap­proval for Fary­dak has nev­er con­vert­ed in­to a full ap­proval.

Vin­cent Ra­jku­mar, a pro­fes­sor and can­cer doc­tor at the Mayo Clin­ic, told End­points News that he was one of the au­thors on one of the panobi­no­s­tat stud­ies, so it’s hard for him to be ob­jec­tive, but he not­ed:

This is a drug that can help a small sub­set of pa­tients. And would be good to have on the mar­ket than not. Most of us are aware of its lim­i­ta­tions so that’s the rea­son its not used much ex­cept for the oc­ca­sion­al cir­cum­stances. So if they let the ac­cel­er­at­ed ap­proval stand, it’s not like the drug will be overused. On the oth­er hand if they with­draw the ap­proval the com­pa­ny may stop man­u­fac­tur­ing and we may lose a drug that helps at least a small sub­set of pa­tients.

Se­cu­ra Bio CMO David Co­han told End­points via email: “1. FARY­DAK, to date, has not had a neg­a­tive con­fir­ma­to­ry tri­al. 2. Se­cu­ra Bio does not yet know what ques­tion(s) the FDA in­tends to ask the ODAC re­gard­ing FARY­DAK.”

In the case of Mar­qi­bo, Talon Ther­a­peu­tics ini­tial­ly won ac­cel­er­at­ed ap­proval for it in 2012 based on lim­it­ed da­ta from a sin­gle Phase II tri­al. Spec­trum Phar­ma­ceu­ti­cals then ac­quired Talon and the drug for about $11 mil­lion in 2013. In 2018, Spec­trum re­port­ed about $5.5 mil­lion in Mar­qi­bo sales, and then pro­ceed­ed to sell the drug a year lat­er with six oth­er can­cer drugs to Au­robindo Phar­ma’s sub­sidiary Ar­cotech for $160 mil­lion up­front.

Mean­while, Talon had ini­tial­ly agreed to sub­mit the re­sults of its con­fir­ma­to­ry tri­al to the FDA more than three years ago, but it re­mains un­known if that sub­mis­sion oc­curred.

The push to re­view these two dan­gling ac­cel­er­at­ed ap­provals comes as the FDA ear­li­er this sum­mer held an­oth­er ODAC meet­ing to re­view six oth­er in­di­ca­tions, four of which the com­mit­tee end­ed up rec­om­mend­ing re­main on the mar­ket. Since then, three in­di­ca­tions have been pulled vol­un­tar­i­ly by the com­pa­nies, and one has gone on to nab a full ap­proval.

Rick Paz­dur’s On­col­o­gy Cen­ter for Ex­cel­lence at FDA al­so ini­ti­at­ed a re­view of the ac­cel­er­at­ed ap­proval path­way about a year ago, while more re­cent­ly, HHS’ in­spec­tor gen­er­al said it al­so will re­view the path­way, fol­low­ing a quick ac­cel­er­at­ed OK for Bio­gen Alzheimer’s drug Aduhelm.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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(Photo courtesy Pfizer)

FDA's vac­cine ad­comm votes al­most unan­i­mous­ly in fa­vor of Pfiz­er's Covid-19 vac­cine for younger chil­dren

The FDA’s Vaccines and Related Biological Products Advisory Committee on Tuesday voted 17-0, with one panelist abstaining, that the benefits of the Pfizer-BioNTech Covid-19 vaccine outweigh the risks for children between the ages of five and 12.

The vote will likely trigger a process that could allow the shots to begin rolling out as early as next week.

The vaccine, which is one-third of the adult Pfizer dose, proved to be about 90% effective in a placebo-controlled trial in which about 1,500 kids in this age range received the vaccine, and only about 12% of those receiving the vaccine had any adverse event. All serious adverse events in the trial were unrelated to the vaccine.

Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

UP­DAT­ED: As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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