Orphan Drugs: In Development

Orphan Drugs in Development

DEVELOPMENT
STATUS

DISEASE

DRUG NAME

SPONSOR

FDA OFFICIAL DESIGNATION*

Acquired Immune
Deficiency
Syndrome
(AIDS)

3'-azido-2',3'
dideoxyuridine

Berlex
(Wayne, NJ)

treatment of AIDS

Phase I

Ampligen~ HEM Pharmaceutical treatment of AIDS Phase II
(Philadelphia, PA)
HIVID~ Hoffmann-La Roche treatment of AIDS Phase III/
zalcitabine (Nutley, NJ) application submitted
Imuthiol Connaught treatment of AIDS Phase II/III
diethyldithiocarbamate (Swiftwater, PA)
Neupogen~ Amgen treatment of patients with AIDS Phase II
filigrastim (Thousand Oaks, CA) who, in addition, are afflicted with
CMV retinitis and are being treated
with ganciclovir
VaxSyn~ HIV-1 MicroGeneSys treatment of AIDS Phase II
gp 160 antigens (Meriden, CT)
(recombinant vaccine)
zalcitabine National Cancer Institute treatment of AIDS Phase I
(Bethesda, MD)

Addiction, Drug LAAM Biometric Research treatment of heroin addicts suitable Phase III
L-alpha-acetyl- Institute for maintenance on opiate agonists
methadol HCL (Arlington, VA)
National Institute on
Drug Abuse
(Bethesda, MD)
Alpha-1 recombinant human Synergen treatment of congenital alpha-1 Phase I/II
Antitrypsin secretory leucocyte (Boulder, CO) antitrypsin deficiency
Deficiency protease inhibitor
Amenorrhea Norditropin~ Novo-Nordisk adjunct for the induction of ovula- Phase III
somatropin (Princeton, NJ) tion in women with infertility due
to hypogonadotropic hypogonadism
or bilateral tubal occlusion or unexplained infertility, who are undergoing in-vivo or in-vitro fertilization
procedures
Amyotrophic branched chain Mount Sinai Medical treatment of amyotrophic lateral Phase II
Lateral Sclerosis amino acids Center sclerosis
(ALS) (New York, NY)
Anemia interleukin-1 alpha, Immunex for hematopoietic potentiation in Phase II
human (Seattle, WA) aplastic anemia
(recombinant)
interleukin-3 Immunex promotion of erythropoiesis in Phase I/II
human (Seattle, WA) Diamond-Blackfan anemia
(recombinant) (congenital pure red cell aplasia)
Leucomax~ Schering-Plough treatment of patients with aplastic Phase III
granulocyte/ (Madison, NJ) anemia
macrophage-colony
stimulating factor
Marogen~ Sterile Chugai-Upjohn treatment of anemia associated with application submitted
Powder (Rosemont, IL) end-stage renal disease (ESRD)
epoetin beta
PROCRIT~ Ortho Biotech treatment of anemia of prematurity Phase II
epoetin alfa (Raritan, NJ) in preterm infants
Angioedema C 1-inhibitor (human) Immuno Clinical prevention of acute attacks of Phase II
vapor heated Research angioedema, including short-term
(New York, NY) prophylaxis for patients requiring
dental or other surgical procedures
*The designation is issued by the FDA's Office of Orphan Products Development while the drug is still in development. The designation
makes the sponsor of the drug eligible for entitlements under the Orphan Drug Act of 1983. The entitlements include seven years of
marketing exclusivity following FDA approval of the drug for the designated use.
Provided as a Public Service by the Pharmaceutical Manufacturers Association.