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Friday February 5, 12:42 pm Eastern Time
Company Press Release
SOURCE: Orphan Medical, Inc.
Orphan Medical Receives FDA
Approval for Busulfex (Busulfan) Injection
MINNEAPOLIS, Feb. 5 /PRNewswire/ -- ORPHAN MEDICAL, INC. (Nasdaq: ORPH -
news) announced today the Food and Drug Administration (FDA) approved the Company's
new drug application (NDA) for Busulfex(TM) (busulfan) Injection for use in combination
with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic
progenitor cell transplantation for chronic myelogenous leukemia (CML).
An oral form of busulfan has been used in the transplant setting for a number of years. Many
authors have cited the need for an intravenous form of busulfan. Orphan Medical has
answered this need, being the only company to successfully bring an intravenous form of
busulfan to the market. In clinical trials, Busulfex demonstrated consistency within
individual patients, as demonstrated by reproducibility of pharmacokinetic parameters
between doses (doses 9 to 13, steady state Cmax). In addition, 93% of the patients attained
an AUC less than the target of 1500 mMol/min, without dose adjustments, which indicates
the consistency of Busulfex (55/59). Busulfex produced profound myelosuppression in all
patients, and engraftment was achieved in 100% (60/60) of evaluable patients. Additionally,
there were no graft failures reported.
The patients included in this allogeneic clinical study (n=61) were undergoing treatment for
a variety of advanced hematologic malignancies, including acute leukemia past first
remission (first or subsequent relapse), with high-risk first remission, or with induction
failure; chronic myelogenous leukemia in chronic phase, accelerated phase, or blast crisis;
primary refractory or resistant relapsed Hodgkin's disease or non-Hodgkin's lymphoma; and
myelodysplastic syndrome. The patient group was high-risk, in that 75% of patients were
transplanted with active disease and nearly half (48%) had previously undergone one or
more of the following treatments: radiation, at least three chemotherapeutic regimens, or a
stem cell transplant. Despite the advanced condition of these patients, the overall mortality
rate through day 100 post-transplant was 13% (8/61). The most common adverse events in
patients treated with Busulfex include profound myelosuppression in 100% of patients,
nausea (97%, mild/moderate in 92%), stomatitis (97%, grade 3 in 26%), vomiting (95%,
all mild/moderate), and anorexia (85%). The type of adverse events seen with Busulfex did
not differ from those seen with oral busulfan in the transplant setting.
''The development of Busulfex represents an important milestone for these patients,'' said
Borje S. Andersson, M.D., Ph.D., professor, Department of Blood and Marrow
Transplantation at The University of Texas M. D. Anderson Cancer Center in Houston.
''We're excited that this new drug will now be available and plan to incorporate Busulfex
into our protocols immediately.''
''The approval of Busulfex is an important step toward building a strong presence in the
oncology market,'' said John Bullion, Orphan Medical CEO. ''This is an exciting approval
for the Company and for the clinicians and patients this product will benefit. We are
aggressively pursuing other opportunities for this important drug. We will seek additional
indications for Busulfex where oral busulfan has been used. We are in discussions with
leading European oncology companies that could help study these indications and market
Busulfex in Europe. We are also awaiting approval of our expedited application in Canada
where, as in the U.S., we will market Busulfex ourselves.''
Busulfex is expected to be commercially available on or around February 22, 1999. To
order Busulfex, call 800-359-4304 or fax 615-833-9581. For questions of a medical
nature, call 888-8ORPHAN (888-867-7426).
Busulfex is an orphan drug that was granted priority (6-month) review by the FDA. A
priority review indicates that this drug may represent a significant advance over currently
available therapy. The approval of the NDA follows review by the Oncologic Drugs
Advisory Committee which unanimously recommended Busulfex for approval during a
January 13, 1999 meeting.
The Busulfex(TM) (busulfan) Injection formulation and parenteral administration of
busulfan in bone marrow transplant and other conditions is covered by United States Patent
numbers 5,430,057 and 5,559,148. Foreign patents are pending.
Orphan Medical, Inc. is dedicated to patients with inadequately treated or uncommon
diseases. To that end, the Company acquires, develops, and markets products of high
medical value within selected strategic therapeutic market segments. Orphan Medical's Web
Site address is orphan.com
For a complete discussion of INDICATIONS and USAGE, CONTRAINDICATIONS,
WARNINGS, PRECAUTIONS, ADVERSE REACTIONS, and OVERDOSAGE, please see
the full prescribing information attached, available through the Company, on the Orphan
Medial Web Site at orphan.com. or toll free at 888-8ORPHAN
(888-867-7426).
The information in this press release may contain forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995. A number of factors
could cause actual results to differ materially from the Company's assumptions and
expectations. These are set forth in the cautionary statements included in Exhibit 99 to
Orphan Medical's most recent Form 10-Q or Form 10-K filed with the Securities and
Exchange Commission. All forward-looking statements are qualified by, and should be
considered in conjunction with, such cautionary statements.
SOURCE: Orphan Medical, Inc. |
