The 2018 break­down on a chart-top­ping set of new drug ap­provals — and the 5 big things we learned from it

2018 will go down as a remarkable year for biopharma R&D, and not just because the industry posted an all-time record high in new drug/biologic approvals. Officially, there were 59 new molecular entities — plus a string of biologics — approved last year. After looking over the full list of each new drug approved by CDER last year, which you can see in all its spectacular detail below, several major themes come into view.


— After 5 years of historic investment in drug R&D and startups, we’re looking at the transformation of a new generation of biotechs into commercial players; no easy task which in itself is forcing some fundamental changes in many of these companies.

— Over the same period, the FDA has changed from a bureaucratic hurdle to active helper. For established drugmakers, the policy at the agency has been to do whatever it can to speed along new drugs. Under Commissioner Scott Gottlieb, that has even meant reversing three earlier rejections. But so far there have been no credible accusations that the open-door, speed-’em-up policy has threatened patients more than it has helped them.

— The rare/orphan disease R&D strategy — the sweet spot for companies looking to score market-moving advances with high-priced drugs in record time — has come full circle in biotech. And the dominant trend will stoke greater demands in 2019 for pricing reform while begging questions about efficiency in marketing.

— Any analysis of R&D productivity has to highlight how each of the top 10 players in the industry performed. And once again we have some stellar successes, some big no-shows and a lingering concern that the sliding overall ROI on R&D remains unsustainable for the group.

— While this annual look at NDAs over the course of the years necessarily focused on FDA approvals — still the industry standard for top global performance — we’ll note that in 2018 the industry truly began to focus on Asian drug OKs. In particular, China has emerged as both a place to develop new drugs, and also strike deals to commercialize the world’s new drugs. Any regulatory strategy that doesn’t include China now is incomplete.

Here comes commercialization risk

Several of the big approvals in 2018 came through for companies that will now start commercialization work for the first time. The marketing class of 2018 includes GW Pharma, with its groundbreaking anti-convulsive meds, the remarkable arrival of RNAi at Alnylam with big advances in play for Agios, Loxo, Ultragenyx, Ionis/Akcea and even little Verastem with its orphaned PI3K drug duvelisib.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Ku­ra co-founder heads to Asian mul­ti-na­tion­al as biotech eyes the goal posts for lead drug

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.

FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.

The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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2019 a 'trans­for­ma­tive year' for phar­ma M&A. Is that a good thing?

Big Pharma keeps getting bigger.

Fueled by the mega-mergers between Bristol-Myers Squibb and Celgene and between Allergan and AbbVie, the industry last year saw $350 billion worth of M&A, according to the new year-end report from the consultants at PwC.  That’s a more than 50% increase on 2018.

“I kind of look at 2019 as a transformational year,” report author Glen Hunzinger told Endpoints News. 

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Drug-drug in­ter­ac­tions: FDA is­sues guid­ance on clin­i­cal, in vit­ro stud­ies

The FDA on Thursday finalized two guidances providing recommendations to drugmakers on evaluating potential drug-drug interactions (DDIs) for new drugs through clinical and in vitro testing.

“Together, the two final guidances describe a systematic risk-based approach to evaluation and communication of DDIs,” the FDA writes.

The two guidances finalize draft versions released in 2017 and have been revised to clarify their scope, provide additional considerations for conducting prospective studies and to explain “when DDI studies are needed for drugs identified as transporter substrates from in vitro studies.” Both guidances have been renamed from their draft versions to reflect an emphasis on investigating the cytochrome P450 (CYP) enzyme and transporter-mediated drug interactions.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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