USFDA Orphan Drug Approvals 1999
Version 1.2
July 10, 2000
Thiru Balasubramaniam
|
Brand Name
|
Panretin
|
Temodar
|
Ellence
|
Aromasin
|
Curosurf
|
INOmax
|
Targretin
|
|
Generic Name
|
alitretinoin
|
temozolomide
|
epirubicin
|
exemestane
|
poractant alfa
|
nitric oxide
|
bexarotene
|
|
Patent
|
No
|
Yes
|
No
|
Yes
|
No
|
Yes
|
Yes
|
|
Indication(s)
|
Topical treatment of cutaneous lesions in
patients with AIDS-related Kaposi's sarcoma.
|
Treatment of adult patients with refractory
anaplastic astrocytoma
|
Component of adjuvant therapy in patients
with evidence of axillary node tumor involvement following resection of
primary breast cancer.
|
Treatment of advanced breast cancer in postmenopausal
women whose disease has progress following tamoxifen therapy.
|
Treatment (rescue) of Respiratory Distress
Syndrome in premature infants.
|
Treatment of term and near-term (>34 weeks)
neonates with hypoxic respiratory
failure associated with clinical or echocardiographic evidence of pulmonary hypertension |
Treatment of cutaneous manifestations of
cutaneouse T-cell lymphoma in patients who refractory to at least one prior
systemic therapy.
|
|
Company
|
Ligand
|
Schering-Plough
|
Pharmacia & Upjohn
|
Pharmacia & Upjohn
|
Dey
|
INO
|
Ligand
|
|
Inventor
|
Edward Lunt et al.
|
Franco Buzzeti et al.
|
Warren Zapol et al.
|
Marcia Dawson et al.
|
|||
|
Assignee
|
Cancer Research Campaign Technology Limited
(London, GB2)
|
Farmitalia Carlo Erba S.r.l.
|
The General Hospital Corporation (Boston,
M.A.)
|
SRI International, La Jolla Cancer Research
Foundation
|
|||
|
Class
|
1PV
|
1PV
|
1PV
|
1SV
|
1SV
|
1PV
|
1PV
|
|
Cost to consumer over completed course treatment
|
$2,400.31
(For 60gm tube: 2 months of treatment- applied twice a day) Panretin is a palliative; treatment duration depends on patient response) |
*Range (Min. and Max. recommended
daily dose based on body surface area):
$29,029 - $72,319 For two years of treatment depending on body size. First figure: 200mg/day-5 consecutive days per 28 day treatment cycle. Second figure: 500mg/day-5 consecutive days per 28 day treatment cycle. (Drugstore.com) |
Range (recommended starting dose:
100 to 120 mg/square meter)
$6000-$7200 (Pharmacia wholesaler cost-cost to consumer is much greater) Treatment for 6 cycles; each cycle is 21 days) |
$1673.67
For 9 months supply (25 mg/day) Median duration of treatment in clinical trials: 8.9 months (Drugstore.com)
|
1.5 mL: $440
3.0 mL: $762 The initial recommended dose is 2.5mL/kg.
|
INOtherapy Service: $125/hour
Minimum duration: 24 hours (first day $3,000) The total charge of INOtherapy Service per
Source: INO |
*Range (Min. and Max. recommended daily
dose based on body surface area): $13,028-$32,571
For one year's supply.
Second figure: 750mg/day |
|
Government Rights
in Patent |
-
|
No
|
-
|
No
|
-
|
Yes
|
No
|
|
Patent Number
|
Not applicable
|
5,260,291
|
Not applicable
|
4,808,616
|
Not applicable
|
5,485,827
|
5,466,861
|
|
Filing of Key Patent Application
|
-
|
18-Oct-91
|
-
|
7-Jul-86
|
-
|
10-Feb-95
|
25-Nov-92
|
|
Patent Approval
|
-
|
9-Nov-93
|
-
|
28-Feb-89
|
-
|
23-Jan-96
|
14-Nov-95
|
|
NDA Filing
|
27-May-98
|
13-Aug-98
|
15-Dec-98
|
21-Dec-98
|
.
|
16-Jun-97
|
23-Jun-99
|
|
NDA Approval
|
2-Feb-99
|
11-Aug-99
|
15-Sep-99
|
21-Oct-99
|
18-Nov-99
|
23-Dec-99
|
29-Dec-99
|
|
Time between Patent Filing and NDA Approval
|
Not applicable
|
7.82 years
|
Not applicable
|
13.30 years
|
Not applicable
|
4.87 years
|
7.10 years
|
|
# of Clinical Trials in
NDA |
2
|
1
|
2
|
3
|
2
|
2
|
2
|
|
# of Patients in each Clinical Trial
|
Study 1: 268 patients
Study 2: 270 patients |
Study: 162 patients
|
MA-5: 716 patients
GFEA: 565 patients |
Missing Data
|
Study 1: 145 patients
Study 2: 357 patients |
NIROS Study: 235 neonates
CINRGI Study: 186 patients |
Missing Data
|
|
Total # of Patients in Clinical Trials
|
538 patients
|
162 patients
|
1281 patients
|
1058 patients
|
502 patients
|
421 patients
|
152 patients
|