Novel Drug Approvals

Novel drug approvals

New drugs approved by the FDA are approved for marketing in the United States, either through the FDA’s traditional approval process or its Accelerated Approval program.

Among all the new drugs that FDA approves every year, a select group of drugs and biological products represent new and innovative treatment therapies. These novel drug products will result in the first clinical applications of the therapies, opening the range of medication options for clinicians.

The table below lists new molecular entities (NMEs) and therapeutic biological products that were recently approved by FDA’s Center for Drug Evaluation and Research (CDER). Every listing has a link to more information about the product.

The table does not contain products approved by the Center for Biologics Evaluation and Research (CBER) such as vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products.

Traditional approval process

The traditional process involves four steps:

1. Laboratory and animal testing
2. Human testing
3. Review of test results data by the Center for Drug Evaluation and Research (CDER)
4. Approval (or denial) by the FDA

The criterion that the FDA uses for all new drug approvals is whether the positive health benefit of the medication will outweigh its known risks. New drugs are also subject to post-approval monitoring by the FDA. Read more about the drug development, investigation, and approval processes.

The traditional approach can take years for a new drug to complete the process from conception to approval.

Because of the usually long approval process, the FDA implemented an Accelerated Approval program to expedite the approval process.

Accelerated Approval program

To qualify for accelerated approval, a new drug must either:

• Treat serious conditions for which there is no current treatment, or
• Show the potential to provide treatment for a life-threatening or otherwise grave condition with a greater benefit than existing therapies.

New drugs approved through this expedited process must conduct post-approval monitoring to confirm the benefits of the treatment.

Read more about the Accelerated Approval program.

Orphan Drug program

The FDA’s Orphan Drug Program was established to encourage the development of drugs for rare diseases that affect fewer than 200,000 people in the U.S.

Because these diseases often lack market incentives due to small patient populations, the FDA offers benefits and protections to make developing these therapies more feasible for pharmaceutical companies.

To qualify:

1. The drug must treat, diagnose, or prevent a disease or condition affecting fewer than 200,000 people in the U.S., or
2. If the population is larger, the sponsor must show there is no reasonable expectation of recovering development costs through U.S. sales.

Read more about the Orphan Drugs program.