Mast Therapeutics Inc (fka MSTX)

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The U.S. FDA Orphan Drug Product Program Page 3
The orphan drug process is essentially a twofold process. A company or an individual can apply for orphan drug designation as described in this section without having an active clinical program. If orphan drug designation is received from the FDA, the sponsor can conduct investigative clinical studies in the United States to support the proposed designation (and thereby be eligible for the U.S. tax credits [if the NDA is eventually approved] and FDA support as designated below). However, the sponsor is not required to do all or any of the clinical development program in the United States as long as the clinical studies generally meet ICH standards and FDA expectations. A drug is not given Orphan Drug Status until an NDA marketing application is approved by the FDA Therapeutic Area Review Division (the second part of the twofold process). Once this marketing approval is obtained, the OOPD certifies the sponsor with regard to tax credits and exclusivity. Thus, from a competitive perspective, the first sponsor to obtain an approved NDA for an orphan designated product and indication obtains the postmarketing tax credits and 7 years of marketing exclusivity.
There is no formal application for an orphan drug designation. However, the regulations identify what needs to be included in a complete signed and dated document.7 Essentially, it is a five- to ten-page document with appropriate literature references appended (to support the incidence statements and can increase the total size of the submission to approximately one volume) and generally includes:
The specific rare disease or condition for which orphan drug designation is being requested.
• Sponsor contact, drug names, and sources.
• A description of the rare disease or condition with a medically plausible rationale for any patient subset type of approach.
• A description of the drug and the scientific rationale for the use of the drug for the rare disease or condition.
• A summary of the regulatory status and marketing history of the drug.
• Documentation (for a treatment indication for the disease or condition) that the drug will
affect fewer than 200,000 people in the United States (prevalence).
• Documentation (for a prevention indication [or a vaccine or diagnostic drug] for the disease or condition) that the drug will affect fewer than 200,000 people in the United States per year (incidence).
• Alternatively, a rationale may be provided for why there is no reasonable expectation that costs of research and development of the drug for the indication can be recovered by sales of the drug in the United States.
Once the request for designation has been received (OOPD will send an acknowledgment of receipt letter), a formal response will take between 1 to 3 months. On notification of granting of an orphan drug designation, the name of the sponsor and the proposed rare disease or condition will published in the Federal Register as part of the public record. After marketing approval,