Orphan Drugs: In Development

DEVELOPMENT
STATUS

DISEASE
Graft Vs. Host
Disease
(GVHD)
(continued)

DRUG NAME
Orthozyme~ CD5 +
CD5-T lymphocyte
immunotoxin

SPONSOR
Xoma
(Berkeley, CA)

FDA OFFICIAL DESIGNATION

treatment of GVHD and/or rejection
in patients who have received bone
marrow transplants

application submitted

ST1-RTA immuno- Sanofi Pharmaceuticals prevention of acute GVHD in Phase II
toxin (SR 44163) (New York, NY) allogeneic bone marrow
transplantation
thalidomide Andrulis Research treatment of GVHD Phase III
(Beltsville, MD)
thalidomide Andrulis Research prevention of GVHD Phase I
(Beltsville, MD)

Growth Hormone
Deficiency

Geref~
sermorelin acetate

Serono Laboratories
(Norwell, MA)

treatment of idiopathic and organic
growth hormone deficiency in
children with growth failure

Phase III

Norditropin~ Novo-Nordisk treatment of growth failure in application submitted
somatropin (Princeton, NJ) children due to inadequate growth
hormone secretion
Saizen~ Serono Laboratories treatment of idiopathic and organic application submitted
somatropin (Norwell, MA) growth hormone deficiency in
for injection children with growth failure
SK&F 110679 SmithKline Beecham for the long-term treatment of in clinical trials
(Philadelphia, PA) children who have growth failure
due to a lack of adequate endogenous
growth hormone secretion
Somatrel Ferring Laboratories diagnostic measure of the capacity Phase III

NG-29

(Suffev, NY)
(Suffern, NY)

of the pituitary gland to release
growth hormone

Hemodialysis, Flolan~ Burroughs Wellcome replacement of heparin in patients application submitted
Heparin epoprostenol (Rsch. Triangle Park, NC) requiring hemodialysis and who are
Replacement at increased risk of hemorrhage
Hemophilia DDAVP Rhone-Poulenc Rorer treatment of mild hemophilia A and Phase III
desmopressin acetate (Collegeville, PA) von Willebrand's disease
KOGENATE~ Miles Inc. prophylaxis and treatment of application submitted
antihemophilic (Berkeley, CA) bleeding in individuals with
factor (BHK) hemophilia A or for prophylaxis
(recombinant FVIII) when surgery is required in
individuals with hemophilia A
Mononine~ Armour Pharmaceutical replacement treatment and prophy- application submitted
monoclonal factor IX (Collegeville, PA) laxis of hemorrhagic complications
of hemophilia B
NovoSevenTM Novo-Nordisk A/S treatment of patients with Phase II
factor VIIa (Copenhagen, Denmark) hemophilia A&B with and without
(recombinant DNA antibodies against factor VIII/IX and
origin) patients with von Willebrand's
disease
Hemorrhagic Virazole ICN Pharmaceuticals treatment of hemorrhagic fever application submitted
Fever ribavirin (Costa Mesa, CA) with renal syndrome
Hepatitis human MAb against Sandoz prophylaxis of hepatitis B Phase II
hepatitis B virus (East Hanover, NJ) reinfection in patients undergoing
liver transplantation secondary to
end-stage chronic hepatitis B
infection
INTRON~ A Schering-Plough treatment of chronic delta hepatitis application submitted
interferon alfa-2b (Madison, NJ)
(recombinant)
Thymosin Alpha 1 Alpha 1 Biomedicals adjunctive treatment of chronic Phase III
(Washington, DC) active hepatitis B
Hyper- Zixoryn Farmacon treatment of hyperbilirubinemia in Phase II
bilirubinemia flumecinol (Ridgefield, CT) newborn infants unresponsive to
phototherapy
Hyper- DCA Peter Stacpoole, M.D. treatment of homozygous familial Phase II
cholesterolemia sodium dichloro- Univ. of Florida hypercholesterolemia
acetate (Gainesville, FL)
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