[Federal Register Volume 59, Number 167 (Tuesday, August 30, 1994)]
[Unknown Section]
[Page 0]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 94-21281]


[[Page Unknown]]

[Federal Register: August 30, 1994]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

 

Notice of Intent to Award A Grant with Time Constraints

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing its 
intention to accept and consider an application for the award of a 
grant with time constraints to the American Drug Development, Inc. 
(ADD). The purpose of the grant is to provide the orphan drug, Sodium 
Phenylbutyrate, to children with three urea cycle disorders 
(deficiencies of carbamyl phosphate synthetase, ornithine 
transcarbamylase (OTC), and argininosuccinic acid synthetase) which, if 
not treated, are fatal to the patient. Competition is limited to ADD 
because ADD is the entity currently able to manufacture and supply 
Sodium Phenylbutyrate for the treatment of these disorders. ADD is also 
in the process of compiling documentation which will be submitted to 
FDA for approval of a new drug application (NDA) for Sodium 
Phenylbutyrate. Until the NDA is approved, this drug is not available 
to patients by any other means other than through a grant to ADD.

ADDRESSEES: An application form is available from, and completed 
applications should be submitted to: Maura C. Stephanos, Office of 
Contracts and Grants Management (HFA-520), Food and Drug 
Administration, Park Bldg., rm. 3-40, 5600 Fishers Lane, Rockville, MD 
20857, 301-443-6170.
    NOTE: Applications hand-carried or commercially delivered should 
be addressed to the Park Bldg., rm. 3-40, 12420 Parklawn Dr., 
Rockville, MD 20857.

FOR FURTHER INFORMATION CONTACT:
    Regarding the administrative and financial management aspects of 
this notice: Maura C. Stephanos (address above).

    Regarding the programmatic aspects of this notice: Carol A. 
Wetmore, Office of Orphan Products Development (HF-35), Food and Drug 
Administration, 5600 Fishers Lane, rm. 8-73, Rockville, MD 20857, 301-
443-4903.

SUPPLEMENTARY INFORMATION: FDA is announcing its intention to accept 
and consider an application for the award of a grant with time 
constraints to ADD, Baltimore, MD. FDA's authority to enter into grants 
for the development of drugs for rare diseases and conditions is set 
out in Section 5 of the Orphan Drug Act (21 U.S.C. 360ee). FDA's 
research program is described in the Catalog of Federal Domestic 
Assistance No. 93.103. Before entering into grants, FDA carefully 
considers the benefits that such grants will provide to the public.
    ADD's application will undergo a peer review in accordance with the 
Public Health Service policies and procedures governing the review of 
grant applications with time constraints.

I. Background

    For the past 8 years, FDA's Office of Orphan Products Development 
(OPD) has funded a clinical research grant for the treatment of urea 
cycle disorders with Sodium Phenylbutyrate. This grant expires 
September 30, 1994. Due to the life-threatening nature of urea cycle 
disorders, it is necessary to have a 3-month supply of the drug on hand 
in case possible problems in the purity and/or availability of the drug 
arise. For this reason, more drugs must be ordered by September 15, 
1994, to ensure an adequate drug supply. ADD is able to immediately 
manufacture and supply the drug for the treatment of these disorders.

II. Mechanism of Support

A. Award Instrument

    Support will be in the form of a grant. In 1994, the FDA estimate 
for this grant award is $ 750,000 (final amount will be negotiated). 
The award will be subject to all policies and requirements that govern 
the research grant programs of the Public Health Service (PHS), 
including the provision of 42 CFR part 52, 45 CFR part 74, and the PHS 
grants policy statement.

B. Length of Support

    The length of support may be for 24 months with no possibility of 
additional noncompetitive support.

III. Reasons for Award With Time Constraints

    FDA believes that there is a compelling need to award a grant to 
ADD based on time constraints because these urea cycle disorders are 
fatal if untreated. Also, FDA believes that ADD is uniquely qualified 
to fulfill the objectives of the proposed grant because it is able to 
immediately manufacture and supply Sodium Phenylbutyrate.

IV. Reporting Requirements

    Program progress reports and financial status reports will be 
required annually, based on the date of award. These reports will be 
due within 30 days after the end of the budget period. A final program 
progress report and financial status report will be due 90 days after 
expiration of the project period of the grant.

V. Smoke-Free Workplace

    PHS strongly encourages all grant recipients to provide a smoke-
free workplace and promote the nonuse of all tobacco products. This is 
consistent with the PHS mission to protect and advance the physical and 
mental health of the American people.

    Dated: August 24, 1994.
William K. Hubbard,
Interim Deputy Commissioner for Policy.
[FR Doc. 94-21281 Filed 8-29-94; 8:45 am]
BILLING CODE 4160-01-F