Can­cer R&D tips the scales at the FDA as ac­tive INDs climb to record high

Patrick Frey

The FDA pe­ri­od­i­cal­ly likes to give it­self a high grade as a top per­former. And like any good bu­reau­cra­cy, it has the num­bers to back it­self up. But a new pre­sen­ta­tion by Patrick Frey, the chief of staff in the Of­fice of New Drugs at the agency, has come up with a se­ries of slides that al­so un­der­scores some im­por­tant trends in drug R&D.

One of the top snap­shots in the slide show high­lights a key fact. As of the end of No­vem­ber, ac­tive INDs hit 7,020 — and that’s a 14-year high sig­ni­fy­ing a steady growth since 2004, when the num­ber was 4,361.

Break­through ther­a­py des­ig­na­tions, with their promise of speedy reg­u­la­to­ry re­spons­es, have proven to be im­mense­ly pop­u­lar. There have been 529 re­quests and 192 were grant­ed. Fifty-one per­cent of that fig­ure on OKs are for on­col­o­gy and hema­tol­ogy — with a big em­pha­sis on blood can­cers — which rep­re­sents the on­col­o­gy di­vi­sion’s de­ci­sion to em­brace BTDs un­der the guid­ance of Richard Paz­dur at a time can­cer drug re­search has boomed.

New FDA com­mis­sion­er Scott Got­tlieb has been en­cour­ag­ing the rest of the agency to do bet­ter on this score, hold­ing up on­col­o­gy as an ex­am­ple of the kind of high per­for­mance he’s af­ter.

I’ll add that the 12% of BTDs for an­tivi­rals is a note­wor­thy num­ber, sig­ni­fy­ing added sup­port at a time of grow­ing need.

Yes­ter­day’s ap­proval of No­vo Nordisk’s semaglu­tide for di­a­betes brought the year-to-date num­ber of OKs to 41. That’s with­in strik­ing dis­tance of the 45 new ap­provals that came through in 2015, which was a cur­rent-era high for an agency that hasn’t seen a big­ger num­ber since 53 new ther­a­pies were ap­proved in 1996. You can track how new drug de­vel­op­ment swooned, up un­til just a few years ago.

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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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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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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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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Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

Mod­er­na says Covid-19 vac­cine boost­er in­creased im­mune re­sponse against vari­ants of con­cern

About a month after announcing their variant-specific Covid-19 vaccine boosters showed promising results in mice, Moderna says it now has some human data to back it up.

Volunteers given a booster shot about six to eight months after receiving their second dose saw increased antibody levels against SARS-CoV-2 and two variants of concern: B.1.351, which was first identified in South Africa, and P.1, first identified in Brazil, the company said on Wednesday.

Re­gen­eron’s gold­en goose Eylea may stave off biosim­i­lar com­pe­ti­tion un­til 2024 or be­yond

Almost 10 years have passed since its first FDA approval and Regeneron’s macular degeneration injection Eylea continues to pile up sales to the tune of about $5 billion per year, or more than half of Regeneron’s annual revenues.

Those billions are not expected to go anywhere anytime soon thanks to competition, even as Novartis subsidiary Sandoz announced Monday that it’s beginning a Phase III trial for an Eylea biosimilar in 460 patients across 20 countries.

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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