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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M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Con­gress lays out its de­mands for FDA in 2023 — on every­thing from ma­chine learn­ing to im­munother­a­pies

As the US House Appropriations committee marks up a bill today to provide the FDA with a 10% budget boost in FY 2023, the committee also offered up a long wish list of topics that it wants the FDA to address.

The list ranges from urging CDER to better use artificial intelligence and machine learning to enhance its review process for supplements or repurposed drug candidates “by leveraging automated data collection, integration, and advanced statistical methods,” to encourage the FDA to publish guidance for cell therapy developers, to increasing staff in key areas.

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FDA+ roundup: Spring reg­u­la­to­ry agen­da looks to fin­ish off Cures im­ple­men­ta­tion; GAO digs in­to med­ical coun­ter­mea­sures

The Biden administration released its Spring regulatory agenda, offering up plans on which proposed and final rulemakings will be coming down the pike in the not-too-distant future.

For the FDA, many of the draft and final rules are repeats, although the agency will look to clear its plate of remaining 21st Century Cures Act obligations, which have been in the works since 2016.

For instance, although Section 3023 of the Cures Act has a Dec. 2019 statutory deadline, the agenda shows the FDA is still hoping to release two proposed rules related to that part of the bill this September.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

FDA un­veils new draft guid­ance to help with oligonu­cleotide ther­a­peu­tics de­vel­op­ment

While oligonucleotides, a wide variety of synthetically modified RNA or RNA/DNA hybrids that bind to a target RNA sequence to alter RNA and/or protein expression, have been winning approvals in recent years (e.g. Novartis’ cholesterol drug Leqvio), the regulatory agency is offering new draft guidance for those looking to follow a similar path.

The non-binding guidance, titled “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics Guidance for Industry” deals with pharmacokinetic, pharmacodynamic, and safety assessments required as part of oligonucleotide therapeutics R&D.

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