The year in new drugs

The year in new drugs

FDA approvals hit a 20-year high in 2017, with cancer and rare-disease drugs dominating the list of new medicines

January 22, 2018 | Volume 96 Issue 4

By Lisa M. Jarvis

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After a dip in 2016, 2017 brought a two-decade peak in drug approvals. The U.S. Food & Drug Administration gave its green light to 46 new molecular entities, the highest number since 1996. Cancer treatments and drugs for rare diseases continued to command a hefty portion of approvals, and many benefited from an agency that seems motivated to streamline development for truly innovative medicines.

Early on in 2017, it was clear the year would bring a bountiful crop of new medicines. By mid-June, FDA had given its nod to as many new molecules as were approved in all of 2016.

The prior year’s meager output meant many big companies were in need of a rebound in productivity. And indeed, for some, 2017 was a salve. AstraZeneca, for example, had no approvals in 2016. It now has three new products in its portfolio: two cancer treatments and an asthma drug.

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Source: FDA. Shown in order of approval during 2017.
* Wholesale acquisition cost is the manufacturer's list price and does not include discounts or rebates.
 

Pfizer, which in 2016 had a lone approval that came only through a midyear acquisition, also scored three new products last year. However, it must share rights to two of them: the cancer immunotherapy Bavencio, which came to Pfizer in 2014 through a multi-billion-dollar deal with Germany’s Merck KGaA, and the diabetes treatment Steglatro, an internally developed drug that in 2013 Pfizer chose to codevelop with U.S.-based Merck & Co.

For others, the drought persisted. Although Bristol-Myers Squibb continued to expand the types of cancer that are treated with its existing immuno-oncology drugs, 2017 was the company’s second year in a row without a new product approval.

The cost of cancer
Despite ongoing scrutiny over health care costs, new oncology treatments continue to carry high price tags.
Sources: Companies, insurers

Peak
New drug approvals were the highest in 21 years.
Sources: FDA

The drug industry’s record-breaking productivity does come with caveats. Although FDA said 33% of the products it approved belonged to new classes of compounds, most drug industry watchers would consider several of them to be older drugs.

For example, FDA’s list of first-in-class drugs includes Emflaza, a decades-old corticosteroid approved for the first time in the U.S. to treat Duchenne muscular dystrophy. A handful of other treatments either are similarly old drugs just now reaching the U.S. market or work against older protein targets now associated with new diseases.

Cancer treatments continued to dominate the list, representing over a quarter of all new molecules approved last year. However, many of the new oncology drugs are not especially unique and work by the same mechanism of action as already marketed drugs. The approval list included two more CDK4/6 inhibitors, two more PD-L1 inhibitors, and more compounds that block the proteins PARP, BTK, and ALK.

The new cancer treatments were the primary beneficiaries of FDA’s effort to speed development of potentially important new drugs. Of the 12 oncology products approved, nine had breakthrough therapy designation (BTD), a status introduced in 2012. FDA has previously described BTD as triggering an all-hands-on-deck approach at the agency to ensure efficient and well-designed clinical programs for drugs that are novel or address an underserved disease.

The program has been growing quickly. Overall, 17 drugs approved in 2017 had BTD status. In 2015, which previously held the recent record for new drug approvals, just 10 products had the special status.

Beyond cancer, anti-infectives and drugs for rare diseases were the other beneficiaries of breakthrough status. According to the Tufts Center for the Study of Drug Development, BTD is significantly shortening drug development timelines. The average time from Investigational New Drug Application—asking FDA to begin clinical trials—to approval letter was 65 months for the 17 BTD drugs approved in 2015 and 2016, compared with 110 months for drugs approved without the status.

“I would say that’s pretty good confirmation that this is another FDA program that seems to be working,” says Christopher-Paul Milne, director of research at the Tufts center. “What has to come next is expansion into other therapeutic areas of need.”

That shortened development time, which in theory should mean lower R&D costs for companies focused on oncology, is not translating into lower prices. All 12 oncology drugs approved last year featured six-figure annual wholesale acquisition costs, the company’s list price before any rebates or discounts.

Although the biotech industry has been energized by the potential for new technologies and therapeutic modalities to tackle difficult-to-treat diseases, small molecules continue to dominate FDA’s docket. Conventional, chemically synthesized drugs represented almost two-thirds of the new molecular entities approved last year.

I would say that’s pretty good confirmation that this is another FDA program that seems to be working.

Christopher-Paul Milne, director of research, Tufts Center for the Study of Drug Development, speaking about FDA’s breakthrough therapy designation (GPCA)

Still, compared with a decade ago, the new drug list last year featured a wider range of modalities, including peptides, enzyme replacement therapies for rare diseases, and an antibody-drug conjugate.

Industry insiders will notice that the tally of 46 does not include the two new therapeutic modalities that garnered the biggest headlines in 2017. Missing are Novartis’s in pharma Kymriah and Gilead Sciences’ Yescarta, both chimeric antigen receptor (CAR) T-cell therapies, a new class of drugs made by reengineering a person’s own T cells to seek and destroy cancer cells. Also absent is Spark Therapeutics’ Luxturna, the first-ever approved gene therapy for a genetic condition. Luxturna treats a rare form of blindness.

By the numbers

46

New molecular entities approved in 2017

22

Approved in 2016

63%

Small molecules approved

12

Cancer drugs approved in 2017

4

Approved in 2016

37%

Drugs with “breakthrough therapy” status

$702,000

Annual price of treatment with BioMarin’s Brineura

Sources: FDA, companies

By the numbers

46

New molecular entities approved in 2017

22

Approved in 2016

63%

Small molecules approved

12

Cancer drugs approved in 2017

4

Approved in 2016

37%

Drugs with “breakthrough therapy” status

$702,000

Annual price of treatment with BioMarin’s Brineura

Sources: FDA, companies

 

C&EN has long tracked FDA’s actions on new molecular entities, and these cellular treatments—the approval of which the agency called “historic”—fall outside that category. Overall, FDA gave the green light to a combined 56 new molecular entities and biologic therapies.

Similarly, the list does not capture another groundbreaking approval that arrived in 2017. In May, FDA green-lighted Merck & Co.’s cancer immunotherapy Keytruda for use in anyone harboring a specific genetic profile.

Keytruda had already been on the market for three years for a variety of cancer types. But last year the agency gave the drug its first “tissue agnostic” approval, meaning that a genetic mutation rather than the location of the cancer—lung or colon, for example—guides use of the treatment.

Soon after coming on as FDA commissioner on May 9, Scott Gottlieb signaled his intention to ease the path for other tissue-agnostic drugs, and he quickly acted: In December, the agency released draft guidance to clarify the clinical development for such treatments.

“When drugs successfully target these molecular mistakes to reverse the effects of different diseases, we need a development pathway that allows the new drug to pursue approval in each of these novel settings on the basis of the molecular marker that the drug targets,” Gottlieb said when the draft guidance was released.

Even as the breadth of modalities and clinical pathways expands, the agency said that every drug in 2017 was approved within the review time frame required by law; many applications actually got the nod before the deadline.

Looking ahead, the agency appears determined to continue partnering with companies to make the development process for innovative drugs even more efficient.

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