Glossary

NDA/BLA Filing

The NDA (New Drug Application) is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S. The data gathered during the animal studies and human clinical trials of an Investigational New Drug (IND) become part of the NDA

The goals of the NDA are to provide enough information to permit FDA reviewer to reach the following key decisions:

  • Whether the drug is safe and effective in its proposed use(s), and whether the benefits of the drug outweigh the risks.
  • Whether the drug’s proposed labeling (package insert) is appropriate, and what it should contain.
  • Whether the methods used in manufacturing the drug and the controls used to maintain the drug’s quality are adequate to preserve the drug’s identity, strength, quality, and purity.

The Biologics License Application (BLA) is a request for permission to introduce, or deliver for introduction, a biologic product.

Phase 1

Consists of testing the drug product in a small number of humans, normally healthy volunteers, to determine preliminary safety and tolerable dose range.

Phase 2

Usually involves studies in a limited patient population to evaluate the effectiveness of the drug product in humans having the disease or medical condition for which the product is indicated, determine dosage tolerance and optimal dosage, and identify possible common adverse effects and safety risks.

Phase 3

Consists of additional controlled testing at multiple clinical sites to establish clinical safety and effectiveness in an expanded patient population of geographically dispersed test sites to evaluate the overall benefit-risk relationship for administering the product and to provide an adequate basis for product labeling.

sNDA/sBLA FILING

To change a label, market a new dosage or strength of a drug, or change the way it manufactures a drug, a company must submit a supplemental new drug application (sNDA).

ADVISORY COMMITTEE

An advisory committee lends credibility to the product review process and provides a forum for public discussion of certain controversial issues. The process helps air issues that do not have simple answers. For specific products, advisory committees consider the available evidence and provide scientific and medical advice on safety, effectiveness, and appropriate use. The committee will generally vote on the efficacy and safety of the product. The FDA, while not bound by the committee decision, tend to generally ( but not always ) follow the advice given by the committee when making its decision on or before the PDUFA date. An Advisory Committee meeting is generally convened approximately 2-4 months prior to the PDUFA date.

PDUFA DATE & PRIORITY REVIEW

The Prescription Drug User Fee Act (PDUFA) date is the target action date for the FDA to make a decision about a new drug application (NDA). This is sometimes referred by investors as the "FDA Approval date". The FDA may approve the product or issue a Complete Response Letter (CRL) if they choose not to approve. The CRL will set forth in detail the specific deficiencies and, where appropriate, the actions necessary to place the application in condition for approval.

For NME (new molecular entity) NDA and original BLA submissions, the time period for the FDA to review the NDA is set at 12 months following filing (10 month review following a 60 day filing period). A minority of products are fortunate to receive priority review, where a decision will be issued in 8 months (6 month review following a 60 day filing period). It is common for a decision under priority review to be issued well BEFORE the priority review date.

Priority Review designation is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.

For a non-NME original NDA the time frame is TEN months following submission and 6 months for a priority review.

For more detailed information read the FDA PDUFA REAUTHORIZATION PERFORMANCEGOALS AND PROCEDURES

BIOSIMILAR TIMELINES

Non NME original NDA?

NDAs for a new formulation, or new dosage, new dose combination or new manufacturer.

BioDueDiligence gives a good summary of the process, or for a more detailed explanation refer to the FDA website.

PDUFA DATE EXTENSIONS

The FDA is permitted to extend the PDUFA date under certain circumstances, but is required to inform the the company if this is so:

1. A major amendment to an original application, efficacy supplement, or resubmission of any of these applications, submitted at any time during the review cycle, may extend the goal date by 3 months. 

2. A major amendment may include, for example, a major new clinical safety/efficacy study report; major re-analysis of previously submitted studies or a submission of a REMS (Risk Evaluation and Mitigation Strategies). 

3. A major amendment to a manufacturing supplement submitted at any time during the review cycle may extend the goal date by two months.

FDA MEETING

The company may schedule a meeting with the FDA. These meetings are called either Type A, B or C meetings:

A Type A meeting is a meeting which is necessary for an otherwise stalled drug development program to proceed (a “critical path” meeting) or to address an important safety issue.

A Type B Meeting is a 1) pre-IND, 2) end of Phase 1,2 or 3 meeting, or 3) a pre-NDA/BLA meeting.

A Type C Meeting is for all other matters.

​ CATALYST DATE

This date is an estimate as to when the specified event will be complete, according to company press releases or estimate completion dates given by clinicaltrials.gov

TICKER

The trading code of the company used for trading.

​ BREAKTHROUGH STATUS

A breakthrough therapy is a drug: 

·         intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and

·         preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug.  All requests for breakthrough therapy designation will be reviewed within 60 days of receipt, and FDA will either grant or deny the request. To date the FDA has granted the status to just under half of all applicants.