2018年度新药评审报告（英文版）_36页_3mb

2018 CDER Drug Therapy Approvals Summary

Core Content

The 2018 annual report from the FDA's Center for Drug Evaluation and Research (CDER) highlights the significant progress made in new drug therapy approvals, emphasizing innovation, impact, predictability, and access to treatments. CDER approved 59 novel drugs, including first-in-class and orphan-designated medications, which offer new therapeutic options for patients with rare diseases, cancer, neurological disorders, and other serious conditions.

Main Points

1. Innovation in Drug Approvals

• CDER used expedited development and review pathways to bring new therapies to market more quickly.
• First-in-class drugs (19 out of 59, or 32%) represent groundbreaking advancements in treatment approaches.
• New formulations, dosage forms, and new uses for existing drugs were also approved, improving patient care and treatment flexibility.

2. Impact on Patients

• Many of the new therapies have the potential to significantly improve quality of life and survival rates for patients with life-threatening or rare diseases.
• Approvals included first-in-class treatments for conditions like Fabry disease, Dravet syndrome, and HIV-1 multidrug resistance.
• New indications for existing drugs, such as the use of a rheumatoid arthritis drug for ulcerative colitis, expanded treatment options.

3. Predictability and Efficiency

• CDER met PDUFA goals, ensuring timely approvals of new therapies.
• Accelerated approval was used for several drugs, allowing patients to access treatments earlier while more data is collected.
• The use of novel endpoints, such as metastasis-free survival, helped streamline the approval process for oncology drugs.

4. Access to Therapies

• Biosimilars were approved, increasing market competition and potentially reducing costs.
• First-cycle approvals were achieved, meaning the U.S. was often the first country to approve new therapies.
• Expanded access programs were used to evaluate safety and efficacy of drugs for patients with no other treatment options.

Key Information

1. Novel Drugs for Rare Diseases (58%)

• 34 of the 59 novel drugs were for rare or orphan diseases, affecting fewer than 200,000 Americans.
• Notable approvals:
  • Crysvita for x-linked hypophosphatemia (XLH)
  • Epidiolex for Lennox-Gastaut syndrome and Dravet syndrome
  • Palynziq for phenylketonuria (PKU)
  • Doptelet and Mulpleta for thrombocytopenia in patients with chronic liver disease
  • Trogarzo for multidrug-resistant HIV-1

2. Infectious Diseases

• Aemcolo (first non-opioid for opioid withdrawal)
• Xerava for hereditary angioedema
• Tavalisse for hereditary angioedema
• Tpoxx for smallpox
• Krintafel for vivax malaria prevention

3. Neurological Disorders

• Ajovy and Emgality for migraine prevention
• Poteligeo for migraine prevention
• Revcovi for hereditary angioedema
• Ultomiris for hereditary angioedema
• Vizimpro for hereditary angioedema

4. Cancer and Blood Disorders

• Copiktra for chronic lymphocytic leukemia (CLL)
• Braftovi for advanced melanoma
• Daurismo for acute myeloid leukemia (AML) in older adults
• Diacomit for seizures in Dravet syndrome
• Libtayo for metastatic cutaneous squamous cell carcinoma
• Lumoxiti for relapsed or refractory hairy cell leukemia
• Lutathera for gastroenteropancreatic neuroendocrine tumors (GEP-NETs)
• Erleada for non-metastatic, castration-resistant prostate cancer
• Tegsedi for hereditary transthyretin-mediated amyloidosis
• Tibsovo for advanced follicular lymphoma
• Xospata for acute myeloid leukemia (AML)
• Tavalisse for hereditary angioedema
• Vitrakvi for non-small cell lung cancer with NTRK gene fusions

5. Women's Health

• Annovera, the first annual vaginal ring contraceptive for women of reproductive age
• Orilissa for endometriosis-related pain

6. Biosimilars

• CDER approved seven biosimilars, contributing to market competition and cost reduction for biological therapies.

7. New and Expanded Uses

• Doptelet and Mulpleta for thrombocytopenia in chronic liver disease
• Epidiolex for seizures in Dravet syndrome
• Firdapse for Lambert-Eaton myasthenic syndrome
• Omegaven for parenteral nutrition-associated cholestasis in children

Conclusion

The 2018 CDER report showcases a strong year of innovation, efficiency, and patient access. The center prioritized safety, efficacy, and predictability in its approval process, while also focusing on rare diseases and novel therapies. These approvals reflect CDER's commitment to advancing health through scientific innovation and regulatory efficiency.