FDA：2021年新药年度报告-26页_594kb

Summary of CDER's New Drug Therapy Approvals in 2021

Core Content

In 2021, the FDA's Center for Drug Evaluation and Research (CDER) approved 50 new drugs, including 27 first-in-class drugs and 26 orphan-designated drugs, marking significant progress in advancing health through innovation. These approvals span various therapeutic areas, including diabetes, rare diseases, infectious diseases, autoimmune disorders, neurological and psychiatric conditions, and cancer. The report highlights the use of expedited development and review pathways, such as Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval, to bring new therapies to patients more quickly while maintaining rigorous safety and efficacy standards.

Main Points

• Innovation and Expedited Pathways: CDER utilized multiple expedited pathways to accelerate drug approvals. These pathways enabled faster review times and increased flexibility in the development process, particularly for drugs targeting serious or life-threatening conditions with limited treatment options.
• First-in-Class Drugs: 27 of the 50 novel drugs were identified as first-in-class, meaning they introduced new mechanisms of action that differ from existing therapies.
• Orphan Drug Approvals: 26 of the 50 novel drugs were designated for rare diseases, providing much-needed treatment options for patients with conditions that previously had few or no available therapies.
• Access and Cost Reduction: The approval of interchangeable biosimilar products increased competition and potentially reduced costs for patients. Additionally, many drugs were approved in the U.S. before any other country, enhancing patient access.
• Rare Disease Treatments: Several new drugs were approved for rare diseases, including:
  • Achondroplasia: First treatment to increase height.
  • Sleeping Sickness (Human African Trypanosomiasis): First all-oral treatment.
  • Paroxysmal Nocturnal Hemoglobinuria: New therapy for this rare blood disease.
  • Polycythemia Vera: Approved treatment for this rare blood disorder.
  • Cystic Fibrosis: Therapy for the most common mutation in children.
• Cancer Therapies: CDER approved multiple new cancer treatments, including:
  • Four new drugs for non-small cell lung cancer.
  • A first-line immunotherapy for esophageal, gastric, and gastroesophageal junction cancers.
  • Therapies for specific subtypes of breast, kidney, and other cancers.
• Infectious Disease Treatments: Notable approvals include:
  • A monthly injectable regimen for HIV-1 treatment.
  • An injectable antiviral drug to prevent HIV-1 infection.
  • A treatment for smallpox, a disease that is no longer endemic but remains a bioweapon concern.
  • A new treatment for post-transplant cytomegalovirus (CMV) infection.
• Autoimmune and Inflammatory Diseases: CDER approved new therapies for:
  • Ulcerative colitis in both adults and children.
  • Systemic lupus erythematosus (SLE) and lupus nephritis.
  • Moderate-to-severe atopic dermatitis and pruritus associated with chronic kidney disease.
• Neurological and Psychiatric Disorders: New approvals include:
  • Two therapies to reverse opioid overdoses.
  • A new treatment for Alzheimer's disease targeting the disease's pathophysiology.
  • Drugs to reduce the frequency of migraine attacks and treat certain neurological conditions.

Key Information

• CDER's novel drug approvals in 2021 averaged 43 per year from 2012 to 2021.
• The use of expedited pathways helped CDER meet PDUFA goals and ensure timely approvals.
• Several drugs were approved for new pediatric populations, expanding treatment options.
• New uses of approved drugs were also highlighted, including expanded pediatric applications and new indications for existing therapies.
• The report emphasizes the importance of biosimilars and interchangeable biosimilars in increasing access and affordability.
• The report also acknowledges the critical role of the Center for Biologics Evaluation and Research (CBER) in approving a COVID-19 vaccine, which was a significant milestone in the fight against the pandemic.

Conclusion

CDER's 2021 drug approvals reflect a commitment to innovation, patient access, and the development of new therapies for a wide range of diseases. The use of expedited pathways and the focus on rare diseases and unmet medical needs demonstrate the agency's dedication to improving public health and advancing treatment options for patients in need.