FDA and Pen­ta­gon forge break­through des­ig­na­tion agree­ment

Fol­low­ing the Pen­ta­gon’s crit­i­cism of the FDA slug­gish­ness in ap­prov­ing a French freeze-dried plas­ma prod­uct, which some have said oth­er coun­tries’ troops have used for years, FDA and the De­part­ment of De­fense’s Of­fice of Health Af­fairs on Tues­day launched a joint pro­gram to pri­or­i­tize the ef­fi­cient de­vel­op­ment of such med­ical prod­ucts.

The frame­work for the pro­gram was es­tab­lished, via H.R.4374, af­ter DoD sought to cre­ate its own re­view path­way where­by it could ap­prove med­ical prod­ucts out­side of FDA’s purview.

FDA and DoD out­lined their new ap­proach to col­lab­o­rat­ing in an ini­tial work plan, which in­cludes treat­ing “all pri­or­i­ty DoD de­vel­op­ment pro­grams as if they had re­ceived break­through des­ig­na­tion” and pro­vid­ing “ex­ten­sive man­u­fac­tur­ing and clin­i­cal ad­vice for pri­or­i­tized prod­ucts,” as well as is­su­ing guid­ance to fa­cil­i­tate the avail­abil­i­ty of prod­ucts need­ed by DoD.

Cur­rent high-pri­or­i­ty DoD prod­uct pro­grams in­clude freeze-dried plas­ma, cold-stored platelets and cry­op­re­served platelets, so the ini­tial phase of the pro­gram will be con­duct­ed by FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search and Health Af­fairs.

CBER se­nior lead­ers will com­plete a re­view of all prod­ucts de­ter­mined by mu­tu­al agree­ment to be a top pri­or­i­ty to DoD by 30 June 2018.

Tom Mc­Caf­fery

CBER will hold for­mal meet­ings with DoD at least semi­an­nu­al­ly to re­view the Pen­ta­gon’s prod­uct de­vel­op­ment pipeline, and the agen­cies will meet at least quar­ter­ly to have a de­tailed dis­cus­sion of the DoD’s high­est-pri­or­i­ty pro­grams. FDA and DoD will al­so hold at least one work­shop this year to dis­cuss as­pects of the sci­en­tif­ic and clin­i­cal de­vel­op­ment of prod­ucts for mil­i­tary per­sons and to in­form the guid­ance.

“This part­ner­ship re­flects the in­valu­able col­lab­o­ra­tion be­tween Health Af­fairs and the FDA to equip U.S. warfight­ers with the best pos­si­ble mil­i­tary med­ical sup­port as we work to achieve a safer, more se­cure world,” said Tom Mc­Caf­fery, act­ing as­sis­tant sec­re­tary of de­fense for Health Af­fairs. “Ex­pe­di­tious ac­cess to life-sav­ing med­ical prod­ucts for U.S. troops on the bat­tle­field is part and par­cel to en­sur­ing our shared pri­or­i­ty of op­er­a­tional readi­ness.”


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

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.

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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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 dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

Robert Spurr, President Salix Pharmaceuticals

Bausch Health’s Sal­ix pi­lots study to shine light on chron­ic liv­er dis­ease and push back on stereo­types

October is both breast cancer awareness and liver disease awareness month. While there’s no doubt which condition draws more attention during the month, Salix wants to change that.

Salix, Bausch Health’s gastroenterology arm, piloted its first chronic liver disease report and physician survey with results out this week aimed at raising awareness and dispelling stereotypes.

While 4.5 million people have chronic liver disease or cirrhosis – which is even more than 3.8 million women diagnosed with breast cancer – the research found chronic liver disease “has not received the attention or level of effort needed for adequate prevention, diagnosis, and standardization of its management.”

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Katie Fanning, Mozart Therapeutics CEO

Mozart Ther­a­peu­tics makes its of­fi­cial de­but, jump­ing in­to the hot Treg R&D field with some big-name in­vestors back­ing it

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few.

Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate led by ARCH.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

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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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