2021's NDA list in­cludes some ex­tra­or­di­nary ac­com­plish­ments in year #2 of the pan­dem­ic

All the big R&D trends are on display in this new list of drug approvals for 2021. Plus one.

Add up everything OK’d from CDER and CBER, and you have 60 new drug approvals for last year, topping the 59 in 2020. That’s a close second to the 64 OKs that came out of the FDA in 2018. The dark days of the early 2000s are a distant memory now, with a host of hungry upstarts promising to make their own entries one day as Big Pharmas double down on innovation.

There’s the new list of small biotechs that nailed down their first drug approvals, expressing nothing but eagerness to get out there and start marketing — something they all have much to learn about. Not all of these drugs are tipping the scales on the commercial side, but that doesn’t mean they aren’t important to the small orphan populations that set this trend in motion more than a decade ago.

In the meantime the list of smaller players making the leap now include biotechs like Apellis and ADC Therapeutics. Kadmon hit, then sold to Sanofi. Can they perform like the analysts have promised? Hopes and forecasts during development are one thing, but as Biogen execs can tell you, sales is an unforgiving, hard-numbers game.

There are the late arrivals in the win column, often showing up at the regulatory finish line well past schedule, sometimes cuffed and bruised by CMC issues that continue to plague the field. The FDA has shown time and again that it isn’t in a forgiving mood over suspect manufacturing problems. That trend looks embedded into the fabric of the drug approval process, for big and small companies alike.

Novartis dealt with that, before getting their approval of Leqvio (inclisiran) and will now put its colossal shoulder behind the commercial wheel in the LDL market.

The major players, of course, tended to dominate the list of potential blockbusters. That’s another industry tradition. So AstraZeneca and Amgen made notable contributions to the list, for breakthrough asthma as well as impressive cancer drugs. AbbVie made its mark with migraines, which is seeing a new wave of therapies enter the market.

As always, sheer grit counts for much of the success. SeaGen’s success with next-gen ADCs is an example of that. AstraZeneca’s R&D staff almost never gives up, but just keeps battling ahead, changing trial designs, persisting. In that sense, finally finishing its world headquarters in Cambridge, UK in 2021 was a fitting symbol for the company’s stubbornness. They may not wow you with speed every time, but they get to the regulatory goal posts.

But amid all the hard work and endurance, let’s not overlook the year’s triumphs, topped by Albert Bourla’s Pfizer. Faced with a pandemic, Pfizer took its partnership with BioNTech to legend status. Its mRNA vaccine provided an instant burst of sales worth tens of billions of dollars. They got there first, breaking development and regulatory barriers, and set up a franchise that will help transform the multinational for the decade ahead. And let’s not forget that they did it in a year in which they brought through Prevnar 20, a next-gen approach that is expected to safeguard one of its biggest franchises.

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The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

The following Q&A has been edited for length and clarity.

John Carroll:

We’ve had a chance to talk a little bit before here about some of the things that you’ve done. Just really remarkably, a lot of the things that you’ve done early in your career puts you in the path with some amazing science that has had an absolutely huge impact in terms of what we’re seeing now on drug development and some of the new technologies that are coming out here, and not only the new technologies, but also some of the most remarkable people ever.

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Jim Wells (UCSF)

An­ti­bod­ies once act­ed on­ly as pro­tein block­ers. Now, sci­en­tists are find­ing new ways to make them pro­tein de­stroy­ers

The first lab-made antibody medicine was approved in 1986 — it bound to an antigen known as CD3 on T cells and was meant to prevent kidney transplant rejection. While antibody technology improved, most antibodies were made as blocking agents, neutering clamps that attacked cells and proteins.

But then scientists got creative with their engineering. They made antibody-drug conjugates, or ADCs for short, which attached toxins or drugs to the antibodies, enabling them to kill cells. Then they made CAR-T therapies, which attached a patient’s T cell to the targeting fragment of an antibody, to destroy cancer cells.

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As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.

Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.