TDA-2018年度新药评审报告（英文版）-2019.2-36页_3mb

2018 FDA Center for Drug Evaluation and Research (CDER) Annual Report Summary

Core Content

The 2018 annual report by the FDA's Center for Drug Evaluation and Research (CDER) outlines the significant drug therapy approvals that occurred during the year, emphasizing innovation, impact, predictability, and access. The report highlights the introduction of 59 novel drugs, new uses for existing drugs, biosimilars, and new formulations, all aimed at improving patient care and advancing medical treatment.

Main Points

Innovation

• CDER approved 59 novel drugs in 2018, including 19 first-in-class drugs, which represent a significant portion (32%) of the approvals.
• These drugs often introduce new mechanisms of action and target previously unmet medical needs, offering breakthrough treatments in various disease areas.
• CDER also approved new formulations and new dosage forms that provide greater convenience and accessibility for patients.

Impact

• The report emphasizes the positive impact of these approvals on patient care, particularly for those with rare diseases, infectious diseases, neurological disorders, cancer, and other chronic conditions.
• Several drugs were approved for first-time use in the U.S., such as the first non-opioid drug for opioid withdrawal symptoms and the first FDA-approved drug derived from marijuana (Epidiolex).

Predictability

• CDER met its PDUFA goals for the year, ensuring timely and predictable drug approvals.
• The use of expedited development and review pathways helped speed up the approval process, allowing important therapies to reach patients faster.

Access

• Biosimilars were approved to increase market competition and patient access.
• CDER approved 7 new biosimilars, contributing to lower costs and more treatment options for patients.
• The report also highlights first-cycle approvals, where drugs were approved in the United States before any other country.

Key Information

New Drug Approvals by Disease Area

• Rare Diseases: 34 of the 59 novel drugs were approved for rare or orphan diseases, representing 58% of all novel approvals. These include treatments for X-linked hypophosphatemia, Fabry disease, phenylketonuria (PKU), and Lambert-Eaton myasthenic syndrome (LEMS).
• Infectious Diseases: CDER approved the first drug to treat smallpox and two new versions of a malaria drug. Additionally, a new single-dose flu treatment and a new antibiotic for nontuberculous mycobacterial lung disease were approved under the LPAD pathway.
• Neurological Disorders: Three new migraine drugs (Aimovig, Ajovy, Emgality) were approved, all CGRP-R antagonists. A new therapy for multiple sclerosis in children was also introduced.
• Women's Health: The first vaginal ring contraceptive for an entire year was approved, as well as a new treatment for endometriosis.
• Cancer and Blood Disorders: A wide range of cancer therapies were approved, including:
  • First therapy for non-metastatic, castration-resistant prostate cancer (Erleada).
  • First monoclonal antibody for HIV-1 (Trogarzo).
  • First treatment for multidrug-resistant HIV-1.
  • New therapies for specific cancers such as ALK+ non-small cell lung cancer (Lorbrena), Hairy Cell Leukemia (Lumoxiti), classical Hodgkin lymphoma (Libtayo), and metastatic cutaneous squamous cell carcinoma (Libtayo).
  • Two new therapies for thrombocytopenia in patients with chronic liver disease (Doptelet and Mulpleta).

Notable Approvals

• Epidiolex: First FDA-approved drug for seizures in Dravet syndrome, and the first drug derived from marijuana.
• Trogarzo: First monoclonal antibody for MDR HIV-1.
• Doptelet and Mulpleta: First drugs approved under the LPAD pathway for thrombocytopenia.
• Crysvita: First drug for X-linked hypophosphatemia.
• Lutathera: First radioactive drug for GEP-NETs (gastroenteropancreatic neuroendocrine tumors).

Conclusion

The 2018 CDER report underscores the commitment to innovation and patient safety, with a focus on expediting approvals, increasing access, and reducing costs through biosimilars and novel drug development. The report also highlights significant advances in treating rare diseases, infectious diseases, neurological disorders, and cancer, reflecting the diversity and importance of the drugs approved.

Appendix Highlights

• Appendix A: Lists all CDER's novel drug approvals by trade name.
• Appendix B: Provides a summary of novel drug designations, including non-proprietary names, dosage forms, and indications.

Summary of Annual Novel Drug Approvals (2009 - 2018)

• CDER approved 59 novel drugs in 2018, up from an average of 33 per year from 2009 to 2017.
• The increase in novel drug approvals indicates a growing focus on innovation and expanding treatment options for patients.