New drug approvals all but screeched to a halt in 2016, with a measly 22 new OKs after a pair of bountiful years. But the FDA wasted no time in 2017, signing off on 12 drugs in the first quarter alone—besting its record for the same period of any year in recent history.
All told, the agency cleared 46 new molecular entities*—surpassing its 2016 total in June—as well as a handful of key biologics, a new shingles vaccine and a pair of CAR-T therapies for blood cancers.
While the FDA itself has pooh-poohed any suggestions that it has grown more lenient, industry watchers aren’t buying it, EP Vantage wrote in its 2018 Preview. In fact, the agency’s “U-turns” on rigorous safety requirements in drugs, such as Eli Lilly’s rheumatoid arthritis drug Olumiant, and its controversial decision to approve Sarepta’s Duchenne drug Exondys 51 last year are helping drive the perception of a “friendly FDA.”
Novartis and AstraZeneca emerged the clear winners, with three approved therapies each. Pfizer, Roche, Valeant and tandem Sanofi and Regeneron each scored two approvals. Gilead also logged two, if you count Yescarta, picked up in its Kite Pharma buyout, and GlaxoSmithKline grabbed one in the form of shingles vaccine Shingrix. Bristol-Myers was the only top 15 pharma company to go without in 2017.
While cancer drugs notched 14 approvals, most of these targeted specific subtypes of cancer. Novartis’ Kisqali and Eli Lilly’s Verzenio, for example, were approved for patients with HR-positive, HER2-negative breast cancer, and Celgene and Agios’ Idhifa is for treating relapsed or refractory acute myeloid leukemia in patients with a particular mutation.
Pfizer and Merck KGaA’s cancer drug Bavencio beat AstraZeneca’s Imfinzi to approval, but its first indication, the rare skin cancer Merkel cell carcinoma, won’t make a dent in its $4 billion peak sales estimate. The partners are hoping to make it past the FDA in a number of other indications and are testing the drug in kidney cancer, ovarian cancer, non-small cell lung cancer and others.
A Duchenne drug took the crown for most controversial nod for the second year running. Marathon Pharmaceuticals came under fire for slapping an $89,000-per-year price tag on the corticosteroid Emflaza, which had already been available overseas for decades for as little as $1,000 annually. The company then looked for a quick payout by offloading the drug to PTC Therapeutics, which is aiming to lower its price to around $35,000 a year.
2017 saw some firsts, including the first FDA-approved drugs for the movement disorder tardive dyskinesia: Neurocrine’s Ingrezza and Teva’s Austedo. And Tesaro’s Zejula sped through FDA review, becoming the only PARP inhibitor that treats all women with recurrent ovarian, fallopian tube or peritoneal cancer, not just women with a BRCA mutation.
Other diseases gained their first new drug in a while: Newron’s Xadago was the first FDA-approved Parkinson’s drug in more than a decade, and Mitsubishi Tanabe’s Radicava is the first new ALS treatment in 22 years.
Novartis’ Kymriah and Kite’s—now Gilead’s—Yescarta made history as the first CAR-T therapies to win approval. Cleared for two different blood cancers, the treatments require a patient’s T cells to be harvested, engineered to fight cancer and then reinfused back into the patient, costing $475,000 and $373,000, respectively. Industry watchers will be paying close attention to their uptake to “evaluate the future value of these and rival therapies,” and to gauge whether Gilead’s $12 billion bet on Kite was worth it, EP Vantage said.
“Huge leaps forward in cancer care with the anti-PD-(L)1 antibodies and the approval of cutting-edge techniques like CAR-T have raised expectations that the sector can keep delivering at pace,” said EP Vantage.
But it cautioned that “excitement” over the long-term gains in up-and-coming fields—such as gene therapy and RNA-based treatments—should be “tempered by realism about the short-term challenges.” Nonetheless, the outlook is positive.
“If you look at the pace of R&D, there has probably never been a better time for a lot of science,” Ben Yeoh, senior portfolio manager at RBC Global Asset Management, said in the EP Vantage report. “We’re curing cancers that we’ve never cured before, we’ve got new drugs for MS, new treatments for diabetes. Costs aside, we are still making progress on human health.”
Without further ado, here's our list of drugs that earned FDA approval in 2017.
* FierceBiotech has historically included only new molecular entities in this report. This year, we have included a few more, including a vaccine, biologics and the CAR-T therapies.