Hi lrb,
My inquiry was with respect to Xerecept. Your insight with respect to a Memantine pivitol trial also raises some interesting questions. As far as Xerecept goes it is clear that it's status as an Orphan Drug is understood.
A PRIOR XERECEPT PRESS RELEASE:
Richmond, California, April 20, 1998 - Neurobiological Technologies, Inc. (OTC-BB symbol: NTII) announced today that it has received notification from the U.S. Food and Drug Administration (FDA) that Xerecept™, the company's synthetic preparation of human Corticotropin-Releasing Factor (CRF), qualifies for orphan drug designation. The company is currently enrolling patients in a randomized, double-blind, positive-controlled Phase II clinical trial to evaluate Xerecept's ability to control neurological symptoms caused by peritumoral brain edema.
Peritumoral brain edema, or swelling in the brain caused by a tumor, is a serious condition affecting approximately 100,000 patients in the U.S. with brain cancer. In these patients, swelling of brain tissue often impairs neurological functioning more than the tumor itself. Symptoms of peritumoral brain edema may include seizures, difficulty in movement, lack of coordination, and impaired mental faculties.
Orphan drug designation provides a company with seven years of market exclusivity after a drug is approved by the FDA for the market, and also makes a company eligible to receive federal monies for clinical research under the Orphan Drug Grant Program. The U.S. Congress enacted the Orphan Drug Act to promote research and development of therapies for rare diseases affecting fewer than 200,000 Americans.
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The implications are less clear. The CDER Handbook does offer some insights into potential pathways for Orphan Drugs which treat serious conditions for which there are no or less than desirable alternative methods of treatment.
FROM THE CDER HANDBOOK: fda.gov
Subpart E in Section 312 of the Code of Federal Regulations establishes procedures to expedite the development, evaluation, and marketing of new therapies intended to treat people with life-threatening and severely-debilitating illnesses, especially where no satisfactory alternatives exist (Federal Register, October 21, 1988).
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Accelerated development/review (Federal Register, April 15, 1992) is a highly specialized mechanism for speeding the development of drugs that promise significant benefit over existing therapy for serious or life-threatening illnesses for which no therapy exists. This process incorporates several novel elements aimed at making sure that rapid development and review is balanced by safeguards to protect both the patients and the integrity of the regulatory process.
Accelerated development/review can be used under two special circumstances: when approval is based on evidence of the product's effect on a "surrogate endpoint," and when the FDA determines that safe use of a product depends on restricting its distribution or use. A surrogate endpoint is a laboratory finding or physical sign that may not be a direct measurement of how a patient feels, functions, or survives, but is still considered likely to predict therapeutic benefit for the patient.
The fundamental element of this process is that the manufacturers must continue testing after approval to demonstrate that the drug indeed provides therapeutic benefit to the patient. If not, the FDA can withdraw the product from the market more easily than usual.
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I guess the big question is does Xerecept meet this standard relative to the alternative existing treatment available for peritumoral brain edema. ie the synthetic corticosteroids with their concomitant serious adverse side-effects. If I were to make an educated guess I would say Xerecept is as good a candidate as any for such consideration provided P-2 results prove to have a high incidence of efficacy.
THE TRUTH IS IT SHOULD BE AN INTERESTING NINE MONTHS
Jeffrey
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