Orphan Drugs: In Development

ORPHAN DRU GS
Presented by the Pharmaceutical Manufacturers Association
1992 Annual Survey

he latest survey by the Pharmaceutical Manufacturers Association on "Orphan Drugs in
Development" shows that 189 medicines
are in development to treat rare diseases.
This compares with 133 orphan drugs in
development in 1989 and 176 in 1991.
Details of the survey results and comparisons with previous years are provided in
the table on this page.
Under the Orphan Drug Act of 1983,
the Food and Drug Administration, as of
March 6, 1992, has granted 488 orphan
drug designations that are currently
active. An orphan drug designation by
FDA, granted before the drug is approved
for marketing, entitles the sponsor to
certain incentives following development
and approval of the drug for the designated rare disease condition. The most
important of these incentives is seven
years of marketing exclusivity following
FDA approval of a drug for the designated use.
Other findings in our survey are:
* Some important areas of orphan
drug research include rare diseases in the
following areas: AIDS and AIDS-related
conditions with 20 drugs in development, cancers with 58, childhood diseases with 50, diseases of women with
17, genetic disorders with 35, and neuromuscular disorders with 15. More details
on the orphan drugs in development in
these areas is contained in a table on
page 2.
* Some of the 189 orphan drugs have
been designated by FDA for more than
one rare condition, resulting in a total of
242 orphan designations. Each designation, along with the name of the drug, its
development status and the drug's sponsor, are listed, beginning on page 3 of
this report. All drugs listed under the
heading "Orphan Drugs in Development"
are either in human clinical trials or have
an application for marketing at the FDA
for review.
* Of the 242 orphan drug designations, 47 are awaiting approval at the
FDA, compared with 50 in 1991 and 40
in 1989. Another 98 are in the final stage

SUMMARY OF SURVEY RESULTS 1989 1991 1992
* Total Active Designations 299 422 488
* Total Approved Medicines 36 54 64
* Total Orphan Drugs in Development 133 176 189
* Total Companies/Sponsors Developing Orphan Drugs 85 120 132
SURVEY RESULTS BY DEVELOPMENT STATUS
* Phase I 15 20 17
* Phase I/II 6 4 10
* Phase II 39 49 61
* Phase II/III 8 14 6
* Phase III 61 86 97
* Phase III Completed 0 0 1
* Phase III/Application Submitted 1 0 1
* Applications Submitted 39 50 46
* Applications Pending 1 0 0
* Clinical Trial Status Unidentified 2 9 3
TOTAL RESEARCH PROJECTS 172 232 242
(Reflects medicines in development for more than one use.)
SURVEY RESULTS BY DISEASE CATEGORY (DRUGS)
* AIDS and AIDS-Related Conditions 18 24 20
* Cancer 37 54 58
* Childhood Diseases 38 46 50
* Diseases of Women 9 12 17
* Genetic Disorders 28 31 35
* Neuromuscular Disorders 10 11 15

of development, compared with 86 in
1991 and 62 in 1989.
* Developing these drugs are 111
companies and 21 other sponsors. Many
of these are smaller companies. Some 47
PMA member companies are involved in
developing 85 of the orphan drugs.
Another 64 orphan drugs have been
approved to treat 74 rare disease conditions. They are listed under "Approved
Orphan Drugs" beginning on page 15.
The seven years of market exclusivity
has expired for 5 of these approved
orphan drugs, as indicated in the listing.
Our PMA Commission on Drugs for
Rare Diseases continues to play an
important role in encouraging orphan
drug development by helping researchers
find corporate sponsors for promising
therapies. Information on the Commission's work and a list of the orphan
drugs for which the Commission is seeking corporate sponsors can be found on

page 23 of this report.
These survey results show the enormous commitment of America's pharmaceutical research companies to finding
treatments and cures for the 5,000 known
rare diseases and the unprecedented progress that they have made in developing
medicines to treat rare diseases. Without
question, the Orphan Drug Act, passed in
1983, has worked exceedingly well in
achieving the important purposes for
which it was enacted-to provide
incentives that would encourage orphan
drug research.
Gerald J. Mossinghoff
President
Pharmaceutical Manufacturers Association

The PMA is grateful to the FDA's Office of Orphan Products Development for their assistance
in producing this survey report and to the 48 organizations that are presenting this information
in cooperation with the PMA. They are listed on the back of this report.