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Home Market News 2017: A year of innovation and advances
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14 Jan 2018

2017: A year of innovation and advances

A summary and highlights of new drug therapy approvals and other drug therapy advances in 2017 by FDA's CDER.

In 2017, FDA's Center for Drug Evaluation and Research's (CDER's) new drug therapy approvals helped a wide range of patients suffering from many different medical conditions to gain new hope for improved quality of life and in some cases, improved chances of surviving life-threatening illnesses.

Rare Diseases

Among many other new approvals to help patients with rare diseases, CDER approved the first new treatment for patients with sickle cell disease in almost 20 years and the first non-blood product to treat patients with hemophilia A with inhibitors. For the first time, a treatment is available for adults diagnosed with giant cell arteritis. CDER also approved a new treatment for Batten disease.

Infectious Diseases

CDER approved a new antibiotic to treat certain types of serious skin infections and another to treat complicated urinary tract infections, including kidney infections. They also approved two new treatments for certain patients with chronic hepatitis C; a new drug to help prevent cytomegalovirus infection in patients who have received a bone marrow transplant; and the first therapy in the US to treat Chagas disease.

Neurological Disorders

Last year was a particularly productive year for approving new therapies for patients with neurological disorders. CDER approved new therapies to treat patients with tardive dyskinesia, myasthenia gravis, Duchenne muscular dystrophy, certain forms of multiple sclerosis, amyotrophic lateral sclerosis, and for Parkinson's disease.

Cancer Therapies

2017 was another strong year for making new cancer therapies available. CDER approved new therapies for certain patients with acute lymphoblastic leukemia; Merkel cell carcinoma; certain forms of relapsed or refractory acute myeloid leukemia; certain forms of lymphoma; recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer; and specific forms of liver, breast, and colorectal cancer. Furthermore, the first cancer treatment based on a genetic feature of a cancer rather than the location of the body where the tumor originated was also approved.

Other Advances

CDER approved a new therapy for decreasing heart risk for patients with diabetes; a new drug to treat patients with moderate-to-severe eczema; and three therapies to treat patients with moderate-to-severe plaque psoriasis. CDER also approved the drug in the US with a sensor embedded in the pill that records that the medication was taken. CDER also worked with pharma manufacturers with facilities that were affected by the 2017 Hurricane Season, to address potential drug shortages.

In 2017, CDER approved a wide variety of drug therapies to improve the health of the American public, including the following:

Novel Drugs

In 2017, CDER approved 46 novel drugs either as NMEs, NDAs or BLAs. Of these 46 novel drugs.

  • 15 were identified as first-in-class; e.g., Dupixent (dupilumab) to treat adults with moderate-to-severe eczema.
  • 18 were approved to treat rare or orphan diseases; e.g., Brineura (cerliponase alfa), a treatment for a specific form of Batten disease.
  • 18 were designated as Fast Track.
  • 17 were designated as breakthrough therapies.
  • 28 were designated Priority Review.
  • 6 were approved under the Accelerated Approval program.

    • In 2017, CDER met its PDUFA goal dates for 100% of the novel drugs approved; it also approved 39 of the 46 novel drugs in the 'first cycle' of review. Thirty six of the 46 novel drugs were approved in the US before receiving approval in any other country.

    New Uses and Expanded Uses of Already FDA-Approved Drugs
  • New uses - notable approvals include, for example, Actemra (tocilizumab), Dysport (abobotulinumtoxinA), Imbruvica (ibrutinib), Opdivo (nivolumab) and Keytruda (pembrolizumab).
  • New populations - notable approvals include, for example, Kalydeco (ivacaftor), Sovaldi (sofosbuvir) and Stelara (ustekinumab).

    • Biosimilars

    In 2017, CDER approved five new biosimilars:

  • Cyltezo (adalimumab-adbm), biosimilar to Humira (adalimumab).
  • Ixifi (infliximab-qbtx), biosimilar to Remicade (infliximab).
  • Mvasi (bevacizumab-awwb), biosimilar to Avastin (bevacizumab).
  • Ogivri (trastuzumab-dkst) biosimilar to Herceptin (trastuzumab).
  • Renflexis (infliximab-abda), also biosimilar to Remicade (infliximab).

    • New Formulations
  • CDER approved three new formulations of already approved opioid pain medications in 2017-- oxycodone, hydrocodone, and morphine sulfate. These new formulations have properties that are intended to deter abuse of these highly addictive medications.

    • New Dosage Forms

    Notable approvals in this category include the following:

  • Esbriet (pirfenidone) 534 mg and 801 mg tablets, new dosage forms of a 267 mg capsule originally approved in 2014 as the first new drug in the US to treat patients with idiopathic pulmonary fibrosis. A typical dose of the originally approved product is two or three 267 mg. capsules three times daily. The higher strength tablets enable taking fewer tablets daily
  • Mydayis (mixed salts of a single-entity amphetamine), a new amphetamine dosage form designed to enable once-daily dosing and provide all-day control for attention deficit hyperactivity disorder.
  • Norvir (ritonavir) oral powder, a new dosage form of the tablet and oral solution forms of Norvir, an HIV protease inhibitor approved in 1996 to be used in combination with other antiretroviral agents for the treatment of HIV-1 infection. The oral powder formulation can be mixed in soft foods and does not contain ethanol or propylene glycol and therefore may be a more palatable and safer alternative for children.

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