PDU­FA VII to ex­pand FDA’s re­al-time on­col­o­gy re­views to sup­ple­ments for all ther­a­peu­tic ar­eas

The FDA’s pop­u­lar pro­gram to speed up its re­views of new on­col­o­gy drugs and sup­ple­men­tal ap­pli­ca­tions by months will be ex­pand­ed in­to a larg­er pro­gram for ef­fi­ca­cy sup­ple­ments across all ther­a­peu­tic ar­eas.

The new pro­gram, to be known as Split Re­al-Time Ap­pli­ca­tion Re­view (STAR), first came to light in the min­utes of a De­cem­ber meet­ing of the pre­mar­ket sub­group work­ing on the ne­go­ti­a­tion of the lat­est it­er­a­tion of the Pre­scrip­tion Drug User Fee Act (PDU­FA VII).

The pro­gram will build off the On­col­o­gy Cen­ter of Ex­cel­lence’s Re­al-Time On­col­o­gy Re­view (RTOR) pro­gram, which first be­gan in 2018 and re­ceives sub­mis­sions about 6 weeks pri­or to the full ap­pli­ca­tion and al­lows the FDA to sign off on ap­provals about 3 months ear­ly, ac­cord­ing to da­ta pub­lished in Jan­u­ary.

In the De­cem­ber meet­ing, FDA sought to make STAR a pi­lot pro­gram that would ex­pand the split sub­mis­sion and re­view of re­quired sec­tions of mar­ket­ing ap­pli­ca­tions to ad­di­tion­al prod­uct types and re­view dis­ci­plines out­side of on­col­o­gy. FDA pro­vid­ed ad­di­tion­al de­tails about re­sources need­ed to es­tab­lish and main­tain STAR.

An­oth­er meet­ing of the same PDU­FA VII sub­group in late Jan­u­ary, de­tails of which were post­ed on Wednes­day, al­so not­ed the draft­ing of com­mit­ment lan­guage that would al­low the STAR pro­gram to speed the re­view of ef­fi­ca­cy sup­ple­ments for prod­uct types in all ther­a­peu­tic ar­eas.

An in­dus­try ne­go­tia­tor who said he could not pro­vide more de­tail, be­cause the PDU­FA VII pack­age still needs to be signed off on by var­i­ous lev­els of HHS, told End­points News that the STAR pro­gram would es­sen­tial­ly ex­pand RTOR to oth­er di­vi­sions and of­fices in FDA’s drug and bi­o­log­ics cen­ters.

Both in­dus­try and FDA pre­vi­ous­ly dis­cussed po­ten­tial cri­te­ria, com­po­nents, and process de­tails for ap­pli­ca­tion sub­mis­sions to be re­viewed un­der STAR. Those de­tails will be­come more ap­par­ent as the com­mit­ment lan­guage is fi­nal­ized.

The cre­ation of a new, wider pro­gram to al­low these ex­pe­dit­ed re­views is a big win for in­dus­try, which has watched RTOR shave up to 6 months off of re­view times for some on­col­o­gy ap­pli­ca­tions, in­clud­ing sup­ple­ments and new mol­e­c­u­lar en­ti­ty ap­provals.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.

Thomas Schall, ChemoCentryx CEO (file photo)

Chemo­Cen­tryx plunges as FDA rais­es ques­tions about rare dis­ease drug ahead of ad­comm

ChemoCentryx’s stock price on Wednesday was cut in half by the release of FDA briefing documents ahead of a Thursday adcomm, raising questions on the company’s clinical data to support avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

ANCA-associated vasculitides (AAV) affect small to medium-size blood vessels that can be fatal in less than a year if left untreated, according to FDA. Only Roche’s Rituxan is currently FDA-approved for the treatment of AAV, while glucocorticoids are approved for the broader indication of vasculitis.

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Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.