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

• Novel drugs, which are often among the more innovative products in the marketplace, and/or help advance clinical care by providing therapies never before marketed in the United States;

• New and expanded uses for already FDA-approved drugs;

• Biosimilars, which are highly similar to already FDAapproved therapeutic biological products. These approvals add consumer choice and marketplace competition;

• New formulations or new manufacturers of already FDA-approved products that can provide advantages over original products, such as being able to take the drug on an empty stomach instead of with food, and;

• New dosage forms that can add value to already FDAapproved drugs, such as chewable tablets for patients unable to swallow pills. This report summarizes these approvals and highlights examples, emphasizing those approvals that offer new and innovative treatments to patients in need.

Novel Drugs

Novel drugs are often innovative products that serve previously unmet medical needs or otherwise significantly help to advance patient treatments. The active ingredient or ingredients in a novel drug have never before been approved in the United States. This report lists all of CDER’s novel drug approvals of 2017 and also discusses those that CDER considers notable advances. In 2017, CDER approved 46 novel drugs, either as new molecular entities (NMEs) under New Drug Applications (NDAs), or as new therapeutic biologics under Biologics License Applications (BLAs).

CDER’s Novel Drug Approvals of 2017

CDER’s novel drug approvals for 2017 are listed below.* See CDER’s Novel Drug Approvals for 2017 on the FDA website for the approval dates, non-proprietary names, and what each drug is used for.

This information is accurate as of December 31, 2017. In rare instances, it may be necessary for FDA to change a drug’s NME designation or the status of its application as a novel BLA. For instance, new information may become available which could lead to a reconsideration of the original designation or status. If changes must be made to a drug’s designation or the status of an application as a novel BLA, the Agency intends to communicate the nature of, and the reason for, any revisions as appropriate. ** Product approved with no trade name.

CDER’s Annual Novel Drug Approvals: 2008 – 2017

In 2017, CDER approved 46 novel drugs. The ten-year graph below shows that from 2008 through 2016, CDER has averaged about 31 novel drug approvals per year.

Impact of Novel Drug Approvals

Many of the novel drugs CDER approved in 2017 are notable for their potential positive impact and unique contributions to quality medical care and patient treatment.

First-in-Class

CDER identified 15 of the 46 novel drugs approved in 2017 (33%) as firstin-class, which is one indicator of the drug’s potential for strong positive impact on the health of the American people. These drugs often have mechanisms of action different from those of existing therapies. Novel drugs approved in 2017 identified as first in class by FDA were: Besponsa, Brineura, Dupixent, Emflaza, Giapreza, Hemlibra, Idhifa, Macrilen, Mepsevii, Ocrevus, Prevymis, Radicava, Rhopressa, Rydapt, and Xermelo.

Examples of notable novel First-in-Class approvals for 2017 include:

• Dupixent (dupilumab) to treat adults with moderate-tosevere eczema (atopic dermatitis), and;

• Ocrevus (ocrelizumab) to treat adults with relapsing forms of multiple sclerosis and primary progressive multiple sclerosis.

Drugs for Rare Diseases

In 2017, 18 of CDER’s 46 novel drugs (39%) were approved to treat rare or "orphan" diseases that affect 200,000 or fewer Americans. Patients with rare diseases often have few or no drugs available to treat their conditions. Novel drugs approved in 2017 with the orphan drug designation were: Aliqopa, Alunbrig, Austedo, Bavencio, benznidazole, Besponsa, Brineura, Calquence, Emflaza, Hemlibra, Idhifa, Macrilen, Mepsevii, Prevymis, Radicava, Rydapt, Xermelo, and Zejula.

Examples of drugs that advance the care of patients with rare diseases approved in 2017 include:

• Brineura (cerliponase alfa), a treatment for a specific form of Batten disease, a rare disease that can cause progressive neurological impairments, including seizures, visual problems/blindness, personality and behaviour changes, dementia, and loss of the ability to walk, talk, and communicate, and;

• Hemlibra (emicizumab), for the prevention of bleeding or to reduce the frequency of bleeding episodes in patients with hemophilia A who have developed antibodies called Factor VIII inhibitors. This is the first non-blood product approved for this condition.

Keep Reading: Other Novel Drug Approvals: Advances in Patient Care Across a Broad Range of Diseases

Written by

Janet Woodcock, M.D.

Director, Center for Drug Evaluation and Research, FDA

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