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GUIDANCE FOR INDUSTRY 
 
SMALL ENTITIES COMPLIANCE GUIDE 
 
Designation of New Animal Drugs for Minor Uses or Minor Species 
 
(This guidance replaces the version dated September 1, 2010.  This guidance updates contact 
information, adds a reference to the first question on page 8, and also reflects minor formatting 
changes.) 
 
Submit comments on this guidance at any time.  Submit written comments to the Division  of 
Dockets Management (HFA-305), Food and Drug Administration,  5630 Fishers Lane, Room 
1061, Rockville,  MD 20852.  Submit electronic comments on the guidance at 
http://www.regulations.gov.   All comments should be identified  with Docket No. FDA-2010-D-
0432. 
 
For further information  regarding this document, contact Stuart Jeffrey, Office of Minor Use 
and Minor Species Animal Drug Development (HFV-50), Center for Veterinary Medicine, 
Food and Drug Administration,  7500 Standish Place, Rockville,  MD 20855, 240-402-0568; 
email:  stuart.jeffrey@fda.hhs.gov. 
 
Additional  copies of this guidance document may be requested from the Communications  Staff 
(HFV-12), Center for Veterinary Medicine, Food and Drug Administration,  7519 Standish Place, 
Rockville,  MD 20855, and may be viewed on the Internet at 
http://www.fda.gov/AnimalVeterinary/GuidanceComplianceEnforcement/GuidanceforIndustry/d
efault.htm or http://www.regulations.gov. 
 
 
U.S. Department of Health and Human Services 
Food and Drug Administration 
Center for Veterinary Medicine 
July 2014 
    Contains Non-Binding Recommendations 
Guidance for Industry 
Small Entities Compliance Guide 
 
Designation of New Animal Drugs for Minor Uses or Minor Species 
 
This guidance represents the Food and Drug Administrations (FDAs) current 
thinking on this topic.  It does not create or confer any rights for or on any person and 
does not operate to bind FDA or the public.  You can use an alternative approach if the 
approach satisfies the requirements of the applicable statutes and regulations.  If you 
want to discuss an alternative approach, contact the FDA staff responsible for 
implementing this guidance.  If you cannot identify the appropriate FDA staff, call the 
appropriate number listed on the title page of this guidance. 
 
 
I.  Background 
 
On July 26, 2007 (72 FR 41010), FDA published  a final rule in the Federal Register, entitled 
Designation  of New Animal Drugs for Minor Uses or Minor Species.  This final rule, which 
implemented  section 573 of the Minor Use and Minor Species Animal Health Act of 2004 
(MUMS act), established new regulatory procedures that provided incentives intended to make 
more drugs legally  available to veterinarians and animal owners for the treatment of minor 
animal species and uncommon diseases in major animal species. These regulations described 
the procedures for designating a new animal drug as a minor use or minor species drug.  Such 
designation establishes eligibility  for the incentives provided by the MUMS act. 
 
FDA has prepared this Small Entities Compliance Guide in accordance with section 212 of the 
Small Business Regulatory Enforcement Fairness Act of 1996 (Public Law 104-121).  This 
document is intended to provide guidance on the requirements of Title 21, Code of Federal 
Regulations,  new Part 516, subpart B. 
 
FDAs guidance documents, including  this guidance, do not establish legally  enforceable 
responsibilities.   Instead, guidances describe the Agencys current thinking  on a topic and 
should be viewed only as recommendations,  unless specific regulatory or statutory 
requirements are cited.  The use of the word should in Agency guidances means that 
something is suggested or recommended, but not required. 
 
II. Purpose 
 
What is Designation? 
 
Designation refers to the status that certain new animal drugs can achieve under the provisions 
of section 573 of the Federal Food, Drug, and Cosmetic Act (FFDCA or the act), which was 
added to the act by the Minor Use and Minor Species Animal Health Act of 2004.  The purpose 
of designation  is to determine eligibility  for incentives for the approval or conditional  approval 
of Minor Use and Minor Species (MUMS) new animal drugs.  In order to be eligible  for 
designation  a new animal drug product must be intended for a minor use in a major species or 
for use in a minor species.  In other words, it must be a MUMS drug.  The major species of 
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animals are horses, dogs, cats, cattle, pigs, turkeys, and chickens.  All other animal species 
(except humans) are minor species (21 CFR 516.3). 
 
III. Questions and Answers 
 
How does designation help get more drugs approved for minor use or minor species? 
 
Designated drugs are eligible  for incentives for the development of MUMS drugs for which 
sponsors might not otherwise pursue approval due to the drugs' limited  market potential. 
 
MUMS designated drugs may obtain exclusive marketing rights for a period of seven (7) years 
after approval or conditional  approval.  The exclusive marketing rights associated with 
designation  prevent the Food and Drug Administrations  (FDA) Center for Veterinary Medicine 
(CVM) from approving or conditionally  approving,  during those seven years, the same drug in 
the same dosage form for the same intended use as a designated product (21 CFR 516.31). 
 
Designation also provides eligibility  to apply for Federal grants to assist in defraying the 
costs of qualified  safety and effectiveness testing associated with the development of a 
new animal drug for a minor use or minor species. 
 
Designation does not change the safety or effectiveness standards for approval or conditional 
approval. 
 
Are all animal drugs intended for minor use in major species or for use in minor species 
eligible for designation? 
 
No.  Under the act, a new animal drug may not be designated if it contains the same drug in 
the same dosage form for the same intended use as a new animal drug that is already 
approved, conditionally  approved, or designated.  Each of these conditions  of sameness is 
described in detail in Title 21 of the Code of Federal Regulations  (CFR) Section 516.3(b).  A 
drug that FDA has found to be functionally  superior is not considered the same as a drug that 
is already approved or conditionally  approved even if it is otherwise the same drug in the same 
dosage form for the same intended use (21 CFR 516.25(a)(3) and 516.3(b)). 
 
Does a MUMS drug have to be designated in order to be eligible for conditional 
approval? 
 
No.  While only MUMS drugs are eligible  for conditional  approval, a MUMS drug does not have 
to be designated in order to be conditionally  approved. 
 
Can foreign firms get drugs designated in the U.S.? 
 
Yes, but foreign firms must have a permanent-resident U.S. agent in accordance with 21 CFR 
516.22. 
   
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How can I determine whether an intended use is a minor use? 
 
In the MUMS provisions  of the act, Congress defined a minor use as the intended use of a drug 
in a major species for an indication  that occurs infrequently  and in only a small number of 
animals, or in limited  geographic areas and in only a small number of animals annually 
(FFDCA  201(pp)) (21 USC 321(pp)).  See 21 CFR 516.3(b) for definitions  of the terms 
limited  geographic area and infrequently. 
 
The agency defined a small number for each major species by regulation  (see 43 FR 43050 
amending 21 CFR 516.3(b)).  Those numbers are:  50,000 horses, 70,000 dogs, 120,000 cats, 
310,000  cattle, 1,450,000  pigs, 14,000,000  turkeys, and 72,000,000  chickens. 
 
To demonstrate that an intended use for a drug is a minor use the sponsor must provide 
supporting  information  to FDA (21 CFR 516.21). 
 
1)  A sponsor must provide an estimate of the total number of animals annually to 
which the drug could be given to treat, prevent, or control the intended disease or 
condition.   Sponsors must include in the estimate animals that would be treated as 
part of a herd or flock treatment.  Sponsors must also submit a list of sources used 
to support the estimate. 
 
2)  This annual animal estimate may be reduced if treatment is only medically  justified 
for a subset of animals.  To take advantage of this reduction a sponsor must 
demonstrate that treatment is only medically  justified  for that subset and submit a 
list of sources used to support the proposed reduction. 
 
How does the designation process work? 
 
A MUMS drug sponsor seeking to gain the incentives associated with designation  needs to 
submit two signed and dated copies of the following  information  to the Office of Minor Use and 
Minor Species Animal Drug Development (OMUMS) (21 CFR 516.20): 
 
1)  a request for designation  that is specific (only one drug in one dosage form for a 
specified intended use); 
 
2)  the name and address of the sponsor, the name of the contact person or U.S. agent, 
the generic name and trade name (if any) of the drug, and the name and address of 
the source of the drug (i.e. the active pharmaceutical ingredient); 
 
3)  a description  of the proposed intended use; 
 
4)  a description  of the drug and dosage form; 
   
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5)  a discussion  of the scientific rationale for the intended use of the drug, including 
reference to all studies and data (positive,  negative, or otherwise) available 
regarding the intended use of the drug; 
 
6)  a specific description of the product development plan for the drug, its dosage 
form, and its intended use; 
 
7)  if intended for a minor use in a major species, documentation  to demonstrate that 
the intended use is a minor use (See above, How can I determine whether an 
intended use is a minor use?); 
 
8)  a statement that the sponsor submitting  the request is the real party in interest of 
the development,  production,  and sale of the drug; 
 
9)  a statement that the sponsor acknowledges that FDA will make certain information 
regarding the designation  publicly  available (for information  that will be made 
publicly  available,  see 21 CFR 516.28). 
 
FDA will refuse to grant a request for MUMS drug designation if (21 CFR 516.25): 
 
1)  the drug is not intended for use in a minor species or FDA determines that there is 
insufficient  evidence to demonstrate that the drug is intended for a minor use in a 
major species; 
 
2)  the drug is the same drug in the same dosage form for the same intended use as one 
that already has a MUMS drug designation; 
 
3)  the drug is the same drug in the same dosage form for the same intended use as one 
that is already approved or conditionally  approved; 
 
4)  the sponsor has failed to provide 
 
a)  a credible scientific rationale in support of the intended use; 
 
b)  information  regarding the product development plan for the drug, its dosage 
form and its intended use sufficient to establish that adherence to the plan can 
lead to successful drug development in a timely manner; 
 
c)  any other information  required by 21 CFR 516.20 (listed above). 
 
A sponsor will only receive market exclusivity  rights when its designated drug is approved or 
conditionally  approved for the same intended use and dosage form as previously  designated.  A 
sponsor can, however, request an amendment to the designated intended use prior to approval or 
conditional  approval.  The request will be granted if FDA determines that 1) the initial 
designation  request was made in good faith, 2) the amendment is a response to unexpected 
research findings,  FDA recommendations,  or other unforeseen developments and 3) for minor 
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use drugs, the change in intended use does not change the minor use status of the drug (21 CFR 
516.26). 
 
Under what conditions will OMUMS terminate a designation? 
 
Once granted, the designation  of a MUMS drug will be terminated (21 CFR 516.29): 
 
1)  upon notification  of FDA by a sponsor of the decision to discontinue  active pursuit 
of approval or conditional  approval; 
 
2)  upon notification  of FDA by a sponsor of its decision to discontinue manufacture 
of an approved or conditionally  approved drug.  The sponsor must notify FDA at 
least one year before it intends to discontinue  manufacturing; 
 
3)  upon the expiration of the 7 years of exclusive marketing rights; 
 
FDA may terminate designation for a drug (21 CFR 516.29): 
 
1)  if FDA determines that the sponsor is not actively pursuing approval or conditional 
approval with due diligence  (e.g., failure to provide annual progress reports); 
 
2)  if FDA determines that a sponsor is unable to provide sufficient quantities of an 
approved or conditionally  approved drug to meet the needs for which it is 
designated (as specified in section 573 of the act and 21 CFR 516.36); 
 
3)  if FDA finds that the request for designation  contained an untrue statement of 
material fact; 
 
4)  if FDA finds that the request for designation  omitted material information; 
 
5)  if FDA finds that the drug, in fact, was not eligible  for designation  at the time of 
submission  of the request (note that when a drug has been designated for a minor 
use, the designation will not be terminated because the number of animals to which 
the drug could be administered subsequently  increases--see 21 CFR 516.29(h)); 
 
6)  if the same drug in the same dosage form for the same intended use is approved or 
conditionally  approved for another sponsor before the MUMS-designated drug is 
approved or conditionally  approved; 
 
7)  if FDA withdraws the approval or conditional  approval of the application  for the 
drug. 
   
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For an approved or conditionally  approved drug, termination  of MUMS drug designation 
terminates the sponsors exclusive marketing rights but does not withdraw the approval or 
conditional  approval of the application  for the drug (21 CFR 516.29(g)).  When a designation 
is terminated, FDA will notify the sponsor in writing and give public notice of the termination 
(21 CFR 516.29(i)). 
 
When does the period of exclusive marketing rights take effect? 
 
The period of exclusive marketing rights for designated MUMS drugs begins on the date of 
approval or conditional  approval as stated in the FDA approval letter and, generally, continues 
for seven years (21 CFR 516.31(a)).  The agency will send the sponsor, or the permanent-
resident U.S. agent, written notice of the initiation  of exclusive marketing rights and also make 
this information  publicly  available (21 CFR 516.34). 
 
As noted above, designation  of an unapproved drug may be terminated if the same drug in the 
same dosage form for the same intended use is approved or conditionally  approved for a 
competing sponsor.  In such case, neither sponsor would be eligible  for the incentives associated 
with designation. 
 
In spite of a grant of exclusive marketing rights, FDA may approve or conditionally  approve the 
same drug for the same dosage form and the same intended use if: 
 
1)  FDA terminates MUMS drug designation  as described above; 
 
2)  FDA withdraws approval or conditional  approval of the application  for the 
designated drug for any reason; 
 
3)  the sponsor of the designated drug provides written consent for FDA to approve or 
conditionally  approve another application  before the expiration  of 7 years; 
 
4)  the sponsor of the designated drug fails to provide sufficient quantities of the drug 
in accordance with section 573 of the act and 21 CFR 516.36. 
 
Is the designation process subject to animal drug user fees? 
 
No.  The designation  process is not subject to the user fees established and implemented 
under the Animal Drug User Fee Act (ADUFA). 
 
See Guidances for Industry #170 and #173 for further information  regarding user fees and user 
fee waivers. 
 
What reports are necessary following designation? 
 
Upon designation  a sponsor must annually submit to OMUMS a brief progress report regarding 
your designated drug.  The first annual report is due within 14 months of the date that 
designation  is granted, and subsequent reports are due annually on the same date that the first 
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report is due.  An annual report must include a brief account of the drug development process, 
including  1) a short summary of the status and results of studies, 2) a description of the 
investigational  plan for the coming year, and 3) a brief discussion of developments that could 
affect the designation  (e.g., scientific findings  indicating  unexpected issues of safety or 
effectiveness) (21 CFR 516.30). 
 
Can a product be designated and indexed at the same time? 
 
Yes.  A sponsor may have a designated product indexed while concurrently pursuing 
approval or conditional  approval. 
 
Indexing is an alternative means to legal marketing of new animal drugs for non- food 
producing  minor species (or for some early life stages of some minor species). See 21 CFR Part 
516, Subpart C--Index of Legally Marketed Unapproved New Animal Drugs for Minor Species 
for more information  on the indexing  process.  Also, see CVM Guidance for Industry #201, 
Small Entities Compliance  Guide - The Index of Legally Marketed Unapproved New Animal 
Drugs for Minor Species.  
 
Who can I call for assistance? 
 
Call the Office of Minor Use & Minor Species Animal Drug Development (OMUMS) at 240-
402-0568.  This Office exists for the sole purpose of facilitating  increased availability  of 
animal drugs intended for minor species or minor use in major species.  The Office is 
responsible for designation,  indexing,  minor use determinations,  and outreach to MUMS 
stakeholders. 
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