FDA beats the averages on approvals

FDA’s Center for Drug Evaluation and Research approves 45 novel new therapies in 2015

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SILVER SPRING, Md.—The new year brought many gifts for patients and clinicians in the form of 45 novel new therapies approved by the Center for Drug Evaluation and Research (CDER) of the U.S. Food and Drug Administration (FDA), significantly more than the average of 28 the agency has approved during the previous nine years of the past decade.
“But far more important than quantity is quality—and the valuable new roles many of these drugs can serve in advancing medical care and the health of patients,” noted Dr. John K. Jenkins, director of the Office of New Drugs at CDER. “During this past year, we approved many new drugs to treat various forms of cancer, including four to treat multiple myeloma, and others to treat lung, skin, breast, brain, colorectal and other cancers. We also approved new drugs to treat heart failure, high cholesterol, cystic fibrosis, and irritable bowel syndrome, as well as the first approved reversal agent for a commonly-used blood thinner. And, for the second consecutive year, we approved more drugs to treat rare diseases than any previous year in our history.”
That last part caught the notice and approval of the National Organization for Rare Disorders (NORD), which noted that FDA approved 21 new orphan drugs to treat rare diseases in 2015, nearly half (47 percent) of all novel new drugs approved for the year.
“The remarkable number of orphan drugs approved by FDA in 2015 underscores the progress we are making in identifying and studying new therapies, the willingness of the investment community to invest in orphan drugs and the continued flexibility shown by the FDA in reviewing applications that often include small studies due to the small number of patients with the disease,” said Peter L. Saltonstall, president and CEO of NORD, which is the primary independent nonprofit organization representing all patients and families affected by rare diseases in the United States.
“At the same time, we must recognize that our patient community faces new challenges in accessing many new therapies, and that the vast majority of the 7,000 identified rare diseases still have no treatment options,” Saltonstall added. “NORD has been the leading advocate for the rare disease community for more than three decades. The progress reflected in so many new therapies re-energizes all of us who are dedicated to improving the lives of people with rare diseases. We look forward to still more approvals in 2016 and beyond.”
But getting back to CDER’s broader work in 2015, FDA’s Jenkins noted that more than one-third of the novel new drugs CDER approved in 2015 were identified by FDA as “first-in-class,” for example, drugs that use a new and unique mechanism of action for treating a medical condition.
Moreover, he noted of the 2015 approvals:
  • More than 40 percent of these new therapies were approved to treat rare (or orphan) diseases that affect 200,000 or fewer Americans, who often have few or no drug treatment options
  • 60 percent of CDER’s novel new approvals for 2015 were designated in one or more categories of Fast Track, Breakthrough, Priority Review or Accelerated Approval—each of these designations helps speed the development and/or approval process to help bring important medications to the market as quickly as possible
  • 64 percent of CDER’s novel new approvals were approved first in the United States before any other country
“As always, compromises were not made in our review standards as we considered the applications as efficiently as possible,” Jenkins asserted. “In short, each of these therapies had to demonstrate that it was safe and effective before being approved. I am pleased and proud to be part of a team that helped bring these new drugs to market as safely as possible. My colleagues and I look forward to another productive year serving the American public.”
“Each year, CDER approves hundreds of new medications, most of which are variations of previously existing products, such as important new dosage forms of already-approved products, or cost-saving generic formulations,” noted Dr. Janet Woodcock, director of CDER. “These new products contribute to quality of care, greater access to medication, more consumer choice, and a competitive marketplace that enhances affordability and public health. However, products in a small subset of these new approvals, that we refer to as novel new drugs, are among the more truly innovative products that often help advance clinical care to another level.”
The novel new drugs approved by CDER in 2015 were Addyi, Alecensa, Aristada, Avycaz, Bridion, Cholbam, Corlanor, Cosentyx, Cotellic, Cresemba, Daklinza, Darzalex, Empliciti, Entresto, Farydak, Genvoya, Ibrance, Kanuma, Kengreal, Kybella, Lenvima, Lonsurf, Natpara, Ninlaro, Nucala, Odomzo, Orkambi, Portrazza, Praluent, Praxbind, Repatha, Rexulti, Savaysa, Strensiq, Tagrisso, Tresiba, Unituxin, Uptravi, Varubi, Veltassa, Viberzi, Vraylar, Xuriden, Yondelis and Zurampic.
Looking specifically once more to the rare disease category, some of the notable new drugs highlighted by CDER in its report on 2015 approval were Kanuma to treat lysosomal acid lipase deficiency; Orkambi for the lung disease cystic fibrosis; Strensiq, a long-term enzyme replacement therapy in patients with infantile- and juvenile-onset hypophosphatasia, a serious and sometimes fatal bone disease; Unituxin to treat pediatric patients with high-risk neuroblastoma; and Xuriden to treat patients with hereditary orotic aciduria, a condition that can result in blood abnormalities (anemia, decreased white blood cell count, decreased neutrophil count), urinary tract obstruction, failure to thrive and developmental delays.
(For more about the 2015 FDA approvals, also see January 2016 issue Editor's Focus piece titled "Still no silver bullet, but progress continues against cancer")

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