FDA commissioner Rob Califf (AP Photo/Jose Luis Magana)

Su­per-speedy can­cer drug re­views: FDA un­veils new draft guid­ance to help spon­sors ap­ply

How does a can­cer drug cross the FDA fin­ish line 3-5 months be­fore its PDU­FA date? That’s where the Re­al-Time On­col­o­gy Re­view comes in.

For the last five years, FDA’s On­col­o­gy Cen­ter of Ex­cel­lence un­der Rick Paz­dur has been qui­et­ly tapped in­to RTOR to al­low spon­sors to pro­vide even ear­li­er, seg­ment­ed sub­mis­sions of crit­i­cal ef­fi­ca­cy and safe­ty da­ta, there­by en­abling these faster eval­u­a­tions of ap­pli­ca­tions.

On Fri­day, OCE pro­vid­ed its first draft guid­ance on the RTOR pro­gram, ex­plain­ing how spon­sors can ap­ply, what to sub­mit if ac­cept­ed, and how RTOR is dif­fer­ent from rolling re­views, in that rolling re­views re­quire com­plete sec­tions (e.g., the com­plete clin­i­cal mod­ule) to be sub­mit­ted pri­or to a com­plete ap­pli­ca­tion sub­mis­sion.

“At the time top-line re­sults of a piv­otal tri­al(s) are avail­able and the data­base has been locked, an ap­pli­cant may ap­ply for re­view un­der RTOR by sub­mit­ting a re­quest via email,” the agency ex­plains. “The ap­pli­cant should in­clude their top-line re­sults and a writ­ten jus­ti­fi­ca­tion ex­plain­ing how their ap­pli­ca­tion demon­strates that it is ap­pro­pri­ate.”

Gen­er­al­ly, about 20 busi­ness days lat­er, the FDA re­view di­vi­sion will de­cide if an ap­pli­ca­tion can be ac­cept­ed.

While many of the drugs that have gone through RTOR so far have shaved sig­nif­i­cant time off their PDU­FA dates, the FDA al­so makes clear in Fri­day’s draft, that “this may not be fea­si­ble for all ap­pli­ca­tions due to spe­cif­ic is­sues that may be iden­ti­fied with the ap­pli­ca­tion or over­all work­load con­sid­er­a­tions.”

Gener­ic Drug­mak­ers and CRLs

An­oth­er hot-but­ton is­sue for the agency is the fact that gener­ic drugs of­ten go through mul­ti­ple cy­cles of re­views be­fore win­ning ap­proval.

To help com­bat these long cy­cles of CRLs and re-sub­mis­sions, the agency on Fri­day re­leased fi­nal guid­ance ex­plain­ing how if an AN­DA ap­pli­cant fails to ful­ly ad­dress all de­fi­cien­cies iden­ti­fied in a CRL, “with­in 1 year af­ter is­suance of the CRL (or take ei­ther of the oth­er two ac­tions pre­scribed by reg­u­la­tion and that are de­scribed above), FDA may con­sid­er this fail­ure to be a re­quest by the ap­pli­cant to with­draw the AN­DA.”

As far as changes from the draft is­sued on Sep­tem­ber 29, 2020, the agency said:

Changes from the draft to the fi­nal guid­ance in­clude adding an ap­pen­dix that pro­vides [nine] ex­am­ples of fac­tors that FDA could con­sid­er as the ba­sis for con­clud­ing that an ap­pli­cant’s re­quest for an ex­ten­sion of time to re­spond to a CRL is rea­son­able. In ad­di­tion, ed­i­to­r­i­al changes were made to im­prove clar­i­ty.

Re­mote Reg­u­la­to­ry As­sess­ments Out­side the Pan­dem­ic

The agency on Fri­day al­so is­sued a new Q&A-based draft guid­ance on re­mote reg­u­la­to­ry as­sess­ments, which have been an im­por­tant re­source for the agency dur­ing the pan­dem­ic, and will con­tin­ue to be of use af­ter the pan­dem­ic.

While the agency ex­plains how the FDA does not con­sid­er an RRA to sat­is­fy the le­gal re­quire­ments of what an in­spec­tion is (i.e. in-per­son as­sess­ment of a fa­cil­i­ty), it says they are still valu­able, par­tic­u­lar­ly as FDA has used them to help with ap­proval de­ci­sions, ver­i­fy cor­rec­tive ac­tions for es­tab­lish­ments with an ac­cept­able com­pli­ance sta­tus, and gain com­pli­ance in­sight in­to es­tab­lish­ments that FDA has been un­able to in­spect.

“The Agency be­lieves that FDA’s use of both vol­un­tary and manda­to­ry RRAs, as ap­plic­a­ble, for all types of FDA-reg­u­lat­ed prod­ucts is in the in­ter­est of the pub­lic health, and the Agency is is­su­ing this guid­ance to pro­vide fur­ther trans­paren­cy to stake­hold­ers about the cir­cum­stances in which RRAs may be used both dur­ing and be­yond the COVID-19 pan­dem­ic and pro­mote greater con­sis­ten­cy in the way RRAs are con­duct­ed,” the draft says.

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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FDA's vac­cine ad­comm to re­view first fe­cal trans­plant to treat C. dif­fi­cile in­fec­tions

Back in 2018, Swiss drugmaker Ferring Pharmaceuticals made a big bet on Minnesota-based Rebiotix, buying up the company for its experimental poop-based drug implant to treat an infection caused by C. difficile, a potentially dangerous bacteria, in a new way.

Four years later, Ferring’s fecal microbiota transplant, dubbed RBX2660 or Rebyota, will face the FDA’s adcomm of outside vaccine experts on Sept. 22, debating whether the agency should license the transplant as a treatment for adults following antibiotic treatment for recurrent C. difficile infection.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Ab­b­Vie sur­veys emo­tion­al im­pact of chron­ic leukemia con­di­tion, finds 'roller coast­er' of emo­tions

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.