2018年度新药评审报告英文版_36页_3mb

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

Core Content

The 2018 annual report from the FDA's Center for Drug Evaluation and Research (CDER) highlights the agency's role in advancing health through innovation and the approval of new drug therapies. It showcases a range of novel drugs, expanded uses of existing medications, biosimilars, and new formulations that significantly improve patient care and treatment options.

Main Points

1. CDER's 2018 Drug Approvals

• CDER approved 59 novel drugs in 2018, including new molecular entities (NMEs) and new therapeutic biologics.
• 34 of these (58%) were approved for rare or "orphan" diseases, addressing significant unmet medical needs.
• 19 of the 59 (32%) were identified as first-in-class drugs, indicating major innovations in treatment approaches.

2. Impact of Approvals

• Many of the new therapies provide first-in-class or first-in-the-U.S. treatments for previously untreatable conditions.
• These approvals offer new options for patients with rare diseases, infectious diseases, neurological disorders, heart and lung conditions, women's health, and cancer.

3. Key Innovations and Approvals

• First-in-class approvals:
  • Galafold (migalastat): First oral treatment for Fabry disease.
  • Lucemyra (lofexidine): First non-opioid drug to reduce opioid withdrawal symptoms.
  • Trogarzo (ibalizumab): First monoclonal antibody for MDR HIV-1.
• Biosimilars:
  • CDER approved seven new biosimilars, increasing competition and reducing costs.
• Expanded uses:
  • Epidiolex (cannabidiol): First FDA-approved drug derived from marijuana, for seizures in rare epilepsies.
  • Doptelet (avatrombopag): First drug approved for thrombocytopenia in patients with chronic liver disease.
  • Daurismo (glasdegib): Approved for AML in older adults or those with comorbidities.
  • Lokelma (sodium zirconium cyclosilicate): First treatment for hyperkalemia in adults.
• New formulations and dosage forms:
  • Xofluza (baloxavir marboxam): First single-dose treatment for influenza.
  • Symdeko (ivacaftor): Approved for cystic fibrosis with specific mutations.
  • Nuzyra (tasimelteon): Approved for non-24-hour sleep-wake disorder.
  • Annovera (vaginal ring): First annual contraceptive for women.
  • Omegaven: First injectable fish oil triglyceride for pediatric patients.

4. Regulatory and Development Innovations

• CDER used expedited pathways to streamline the approval process for many therapies.
• New endpoints were used in oncology, such as metastasis-free survival, to evaluate drug efficacy.
• Expanded access programs were leveraged to support the approval of therapies for rare diseases with no existing FDA-approved treatments.

5. Approvals for Specific Conditions

• Rare Diseases:
  • Crysvita: First treatment for XLH (x-linked hypophosphatemia).
  • Asparlas: First treatment for adult patients with a rare form of rickets.
  • Onpattro: First FDA-approved RNAi therapeutic for hereditary transthyretin-mediated amyloidosis.
• Infectious Diseases:
  • Trogarzo: First monoclonal antibody for MDR HIV-1.
  • Krintafel: First one-dose treatment for vivax malaria.
• Neurological Disorders:
  • Ilumya (tildrakizumab): Approved for psoriasis and psoriatic arthritis.
  • Poteligeo (efgartigimod): First therapy for antibody-mediated autoimmune diseases.
• Women's Health:
  • Annovera: First annual contraceptive ring.
• Cancer and Blood Disorders:
  • Copiktra (duvelisib): Approved for CLL/SLL.
  • Lutathera: First radiopharmaceutical for GEP-NETs.
  • Lorbrena: First treatment for ALK+ NSCLC with resistance.
  • Erleada: First treatment for non-metastatic, castration-resistant prostate cancer.
  • Tavalisse (lifitegrast): First treatment for dry eye disease.
• Other Notable Approvals:
  • Xerava (daptomycin): First drug approved under the Limited Population Pathway for Antibacterial Drugs.
  • Vizimpro (sacubanib): First drug to treat a rare form of eye disease (neurotrophic keratitis).

Key Information

• CDER prioritizes safety in all approvals and provides detailed information in the Drugs@FDA database.
• Predictability was maintained by meeting PDUFA goals and completing approvals by or before their target dates.
• Access was enhanced through the approval of new drugs and biosimilars, increasing patient choice and potentially lowering costs.
• The report emphasizes the use of innovative regulatory tools to expedite the approval of therapies, especially for rare and complex diseases.

Conclusion

The 2018 CDER annual report demonstrates the FDA's commitment to innovation, safety, and improving patient access to effective treatments. It highlights the approval of numerous first-in-class and orphan-designated drugs, as well as the use of novel endpoints and development pathways to bring life-saving therapies to market more quickly. These approvals reflect a broader effort to address unmet medical needs and support advancements in a wide range of diseases.