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viPedibiliess F S 2 Advancing Health Through Innovation Table of Contents Introduction . 3 2018: Another Strong Year for Innovation and Advances . 4 Novel Drugs . 7 Impact of Novel Drug Approvals . 9 Innovation: Frequent Use of Expedited Development and Review Pathways . 18 Predictability: Meeting PDUFA Goals . 20 Access: First Cycle Approval and Approvals Compared to Other Countries . 21 New and Expanded Uses of Already FDA-Approved Drugs . 22 New Uses . 22 New Populations . 25 Additional Approvals . 26 Biosimilars . 26 New Formulations and Other Notable Approvals . 28 New Dosage Forms . 30 Conclusion . 31 Appendix A: Drug Designation Summary . 32 Appendix B: Novel Drug Designation Summary. 342018 New Drug Therapy Approvals 3 Introduction Welcome to the FDAs Center for Drug Evaluation and Researchs (CDER) annual report, Advancing Health Through Innovation: New Drug Therapy Approvals. This report helps illustrate CDERs role in bringing innovative new drug therapies that are safe and effective to patients in need. Many of this years new drug therapies are novel drugs - those never before approved or marketed in the United States. Novel drugs often represent important new therapies for advancing patient care. Our report also highlights medically significant approvals for existing drugs. As in past years, many important advances in 2018 use an already FDA-approved drug to treat a new disease or a new population of patients, such as children. Our report emphasizes some of the many innovative ways we were able to evaluate safety and efficacy for these new therapies, as well as key regulatory tools we used to enhance our efficiency and expedite the review and approval of applications. This report also highlights the years biosimilar approvals. Biosimilars have great potential for both patients and the entire health care system. As patents and exclusivity protections for biologics expire in the United States, we can expect many more biosimilars to be submitted for approval. More products on the market means greater competition that can lead to increased access to therapies and lower costs to patients. The decisions we made on these approvals were generally completed by or before their goal dates as defined by Congressionally-approved agreements with industry (referred to as user fee programs). Most were approved in the United States before any other country in the world. Throughout all of our approval evaluations, safety remains our top priority. Our annual Drug Safety Priorities report provides a valuable overview of the many ways we work to ensure safety for all of our approvals. Keep in mind that this report focuses on CDER approvals. FDAs Center for Biologics Evaluation and Research (CBER) also approves many important therapies to advance and protect the health of the American public. For more information, please visit CBERs web page for 2018 Biological Approvals. We trust this report will continue to promote greater understanding of the many ways CDER works to support innovation and improve treatments for patients. Janet Woodcock, M.D. Director, Center for Drug Evaluation and Research 2018 New Drug Therapy Approvals4 Advancing Health Through Innovation 2018: Another Strong Y ear for Innovation and Advances In 2018, FDAs Center for Drug Evaluation and Researchs (CDERs) new drug therapy approvals helped a wide range of patients suffering from many different medical conditions gain new hope for improved quality of life, and in some cases, improved chances of surviving life- threatening illnesses. Rare Diseases Among 34 novel approvals to help patients with rare diseases, CDER approved the first drug to treat patients with a rare, inherited form of rickets, a condition that leads to impaired bone growth and development. Also, CDER approved the first orally-administered drug to treat Fabry disease, a rare and serious disorder that can cause many adverse symptoms, including pain and burning in the hands and feet, and damage to the kidneys and heart. We also approved a new drug to treat patients with phenylketonuria (PKU), a rare dietary condition in which patients are born with an inability to break down protein-containing foods and certain sweeteners, and which can lead to brain and nerve damage. CDER approved many new treatment options for patients in need.5 2018 New Drug Therapy Approvals Infectious Diseases CDER approved the first drug ever to treat smallpox and therefore help in the event of a bioterror attack with this virus. We also approved the first of a new class of drugs to treat patients with HIV-1 infection who have failed other therapy. Additionally, CDER approved two new versions of the same drug for malaria: one to prevent relapse of a certain form, and one to protect travelers to endemic areas from contracting it. CDER also approved a new single dose treatment for influenza (flu). CDER also approved a new formulation of an antibiotic to treat certain patients with nontuberculous mycobacterial lung disease. This is the first antibacterial drug product approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD) pathway, which is designed to streamline development and approval of antibacterial drugs to treat serious or life-threatening infections in a limited population of patients with unmet need. Neurological Disorders CDER approved a new drug to treat seizures in patients with the rare diseases Lennox-Gastaut syndrome and Dravet syndrome. We also approved a second drug to treat seizures in patients with Dravet syndrome. Previously there had been no FDA-approved therapy for use in Dravet syndrome. We approved three new drugs - all in a new drug class - to treat patients with migraine. We also approved two new drugs to treat polyneuropathy (nerve damage) in patients with hereditary transthyretin-mediated amyloidosis, a severely disabling condition that can cause symptoms such as muscle weakness and loss of sensation. Additionally, CDER approved the first therapy to treat multiple sclerosis in children. Heart, Lung, and Circulatory Diseases CDER approved a new drug for the treatment of patients with hereditary angioedema, a disorder that results in recurrent attacks of severe swelling - most commonly affecting the arms, legs, face, intestinal tract, and airway. Womens Health CDER approved the first new treatment in more than 10 years for patients with pain caused by endometriosis, a common condition that affects as many as one in 10 women of child-bearing age, in which the tissue that lines the uterus grows in other parts of the body. Many times, this pain occurs during menstruation. We also approved the first vaginal ring contraceptive that can be used for an entire year. Cancer and Blood Disorders 2018 was another strong year for making new cancer and blood therapies available to patients in need. We approved new advances for certain patients with breast cancer, a new drug to treat certain patients with prostate cancer, and a new drug for patients with a certain type of lung cancer. We also approved: Two previously-approved melanoma (skin cancer) drugs, which are given together to treat patients with a highly aggressive form of thyroid cancer; A new drug to treat patients with an unusual type of cancer that may occur in the pancreas or gastrointestinal tract (neuroendocrine tumors) that is no longer responding to hormone treatment; The first FDA-approved drug for the treatment of patients with pheochromocytoma or paraganglioma (rare tumors of the adrenal gland);6 Novel Drug Approvals Cancer and Blood Disorders (Cont.) Another new cancer therapy that can be used to treat any kind of tumor that has a specific genetic marker, as opposed to where in the body the tumor originated - only the second cancer therapy approved by the FDA to target treatment based on a specific characteristic of a tumor instead of its site of origin; Two new drugs to treat certain adult patients with relapsed or refractory acute myeloid leukemia (a type of cancer of the blood and bone narrow), each to treat patients who have a specific genetic mutation that enables them to be a candidate for treatment with the drug; A new front-line treatment (prior to a relapse or failed treatment with another drug) for patients with acute myeloid leukemia (AML); A new therapy to treat patients with classical Hodgkin lymphoma (a type of cancer of a part of the immune system called the lymphatic system); A new therapy for certain patients with a form of acute lymphoblastic leukemia; A new treatment for adult patients with relapsed or refractory mycosis fungoides (MF) or Szary syndrome (SS), both a type of non-Hodgkin lymphoma in which a type of white blood cell in the body becomes cancerous and affect the skin, and; The first two therapies for thrombocytopenia (a deficiency of platelets in the blood) in patients with chronic liver disease scheduled to undergo a medical or dental procedure. Other Advances CDER approved the first non-opioid drug product approved to help adult patients suffering from opioid withdrawal symptoms. We also approved a new indication for a non-opioid nerve block therapy to help adult patients with pain management for 48 hours after shoulder surgery. Additionally, CDER approved a drug originally approved to treat rheumatoid arthritis to now also treat patients with ulcerative colitis. CDER also approved a new drug therapy for patients suffering from a rare disease affecting the cornea of the eye, called neurotrophic keratitis. It provides a drug therapy alternative to surgery and is considered an important advance because it offers complete corneal healing for many patients with the condition. We also approved seven new biosimilars, which will further help to create competition, increase patient access, and potentially reduce the cost of important biological drug therapies. CDERs drug therapy approvals of 2018 will help advance patient care in many new areas.7 Novel Drug Approvals CDERs Drug Therapy Approvals of 2018 In 2018, 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 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 FDA- approved therapeutic biological products. These approvals add consumer choice and spur 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 FDA- approved 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 otherwtise 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 CDERs novel drug approvals of 2018 and also discusses those that CDER considers notable advances. In 2018, CDER approved 59 novel drugs, either as new molecular entities (NMEs) under New Drug Applications (NDAs), or as new therapeutic biologics under Biologics License Applications (BLAs). As with all FDA- approved products, the new drug therapies discussed in this report are associated with risks. For more information about these drugs and for complete risk information, see the drugs approval letters and FDA-approved labeling at DrugsFDA.8 Novel Drug Approvals CDERs Novel Drug Approvals of 2018 CDERs novel drug approvals for 2018 are listed alphabetically below by trade name.* See Appendix A in this report or visit online, CDERs Novel Drug Approvals for 2018 for the non-proprietary names, dosage forms, and what each drug is used for. Mo * This information is accurate as of December 31, 2018. In rare instances, it may be necessary for FDA to change a drugs 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 drugs 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 CDERs Annual Novel Drug Approvals: 2009 - 2018 In 2018, CDER approved 59 novel drugs. The 10-year graph below shows that from 2009 through 2017, CDER has averaged about 33 novel drug approvals per year. Aemcolo Aimovig Ajovy Akynzeo, Annovera Asparlas Biktarvy Braftovi Copiktra Crysvita Daurismo Diacomit Doptelet Elzonris Emgality Epidiolex Erleada Firdapse Galafold Gamifant Ilumya Krintafel Libtayo Lokelma Lorbrena Lucemyra Lumoxiti Lutathera Mektovi Motegrity moxidectin* Mulpleta Nuzyra Olumiant Omegaven Onpattro Orilissa Oxervate Palynziq Pifeltro Pot
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