Approval is being sought for use as a high-dose conditioning treatment
prior to stem cell transplantation in multiple myeloma and for the
palliative treatment of patients with multiple myeloma for whom oral
therapy is not appropriate.
The Company expects FDA review to take approximately 10 months.
The Company plans to launch this drug with its existing
hematology/oncology sales force next year pending approval.
Our formulation is free of propylene glycol and does not use a custom
solvent for its reconstitution; it is more stable with a longer use
time, simplifying clinical administration logistics.
HENDERSON, Nev.--(BUSINESS WIRE)--
Spectrum Pharmaceuticals (Nasdaq: SPPI), a biotechnology company
with fully integrated commercial and drug development operations with a
primary focus in Hematology and Oncology, today announced the company
has submitted a New Drug Application (NDA) to the U.S. Food and Drug
Administration (FDA) for the approval of Captisol-Enabled Melphalan
(CE-Melphalan) HCl for injection (propylene glycol-free) for use as a
high-dose conditioning treatment prior to hematopoietic progenitor
(stem) cell transplantation in patients with multiple myeloma. Spectrum
is also seeking approval for the palliative treatment of patients with
multiple myeloma for whom oral therapy is not appropriate.
"We believe the lack of propylene glycol in the preparation of
CE-Melphalan eliminates the risk of the toxicities associated with that
excipient," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive
Officer of Spectrum Pharmaceuticals. "The improved solubility and
stability of this novel melphalan formulation, CE-Melphalan, will make
it an attractive treatment option for both transplant conditioning, and
the palliative treatment of patients with multiple myeloma who cannot
take oral melphalan. The enhanced stability of this new CE-Melphalan
formulation simplifies clinical administration and is anticipated to
allow for longer use and infusion times. This NDA submission represents
another important step forward in bringing new treatment options to
The Phase 2 pivotal trial evaluating CE-Melphalan was a multi-center
trial evaluating safety and efficacy. The primary objective of the study
was to determine the overall safety and toxicity profile in multiple
myeloma patients receiving 200 mg/m2 of CE-Melphalan as
myeloablative therapy prior to autologous stem cell transplantation
(ASCT). The secondary objectives evaluated the efficacy of CE-Melphalan
in this patient population as measured by Multiple Myeloma Response Rate
(according to International Myeloma Working Group [IMWG] criteria), and
the rates of myeloablation and engraftment. The primary as well as
secondary endpoints of this Phase 2 trial were met, and a comprehensive
505(b)(2) NDA was submitted to the FDA.
In a previous clinical study, CE-Melphalan met the requirements for
establishment of bioequivalence to the currently approved commercial
intravenous formulation of melphalan. When approved, this demonstrated
bioequivalence of CE-Melphalan should facilitate the use of this new
more stable, propylene glycol-free Captisol formulation of melphalan.
Spectrum Pharmaceuticals gained global development and commercialization
rights to CE-Melphalan from Ligand Pharmaceuticals Incorporated (NASDAQ:
LGND) in March 2013. Spectrum assumed the responsibility for the pivotal
clinical trial and was responsible for filing the NDA. Under the license
agreement, Ligand received a license fee and is eligible to receive
milestone payments, as well as royalties following potential
About Spectrum Pharmaceuticals, Inc.
Spectrum Pharmaceuticals is a leading biotechnology company focused on
acquiring, developing, and commercializing drug products, with a primary
focus in oncology and hematology. Spectrum and its affiliates market
five oncology drugs─ FUSILEV® (levoleucovorin) for Injection in the
U.S.; FOLOTYN® (pralatrexate injection), also marketed in the U.S.;
ZEVALIN® (ibritumomab tiuxetan) Injection for intravenous use, for which
the Company has worldwide marketing rights; MARQIBO® (vinCRIStine
sulfate LIPOSOME injection) for intravenous infusion, for which the
Company has worldwide marketing rights and BELEODAQ™ (belinostat)
for Injection in the U.S. Spectrum's strong track record in in-licensing
and acquiring differentiated drugs, and expertise in clinical
development have generated a robust, diversified, and growing pipeline
of product candidates in advanced-stage Phase 2 and Phase 3 studies.
More information on Spectrum is available at www.sppirx.com.
About Captisol-Enabled Melphalan
Captisol-Enabled, Propylene Glycol-free Melphalan is a novel intravenous
formulation of melphalan being investigated for the multiple myeloma
transplant setting, for which it has been granted an Orphan Drug
Designation by the FDA. This formulation eliminates the need to use
propylene glycol containing custom diluent, which has been reported to
cause renal and cardiac side effects, which in turn limit the ability to
deliver higher doses of therapeutic compounds. The use of the Captisol®
technology to reformulate melphalan also improves its stability and is
anticipated to allow for slower infusion rates and longer administration
durations, potentially enabling clinicians to safely achieve a higher
dose intensity for pre-transplant chemotherapy.
Captisol is a patent-protected, chemically modified cyclodextrin with a
structure designed to optimize the solubility and stability of drugs.
Captisol was invented and initially developed by scientists in the
laboratories of Dr. Valentino Stella at the University of Kansas'
Higuchi Biosciences Center for specific use in drug development and
formulation. This unique technology has enabled six FDA-approved
products, including Onyx Pharmaceuticals' Kyprolis®, Baxter
International's Nexterone® and Merck's NOXAFIL IV. There are
also more than 30 Captisol-enabled products currently in clinical
Forward-looking statement — This press release may contain
forward-looking statements regarding future events and the future
performance of Spectrum Pharmaceuticals that involve risks and
uncertainties that could cause actual results to differ materially. These
statements are based on management's current beliefs and expectations.
These statements include, but are not limited to, statements that
relate to our business and its future, including certain company
milestones, Spectrum's ability to identify, acquire, develop and
commercialize a broad and diverse pipeline of late-stage clinical and
commercial products, leveraging the expertise of partners and employees
around the world to assist us in the execution of our strategy, and any
statements that relate to the intent, belief, plans or expectations of
Spectrum or its management, or that are not a statement of historical
fact. Risks that could cause actual results to differ include the
possibility that our existing and new drug candidates may not prove safe
or effective, the possibility that our existing and new applications to
the FDA and other regulatory agencies may not receive approval in a
timely manner or at all, the possibility that our existing and new drug
candidates, if approved, may not be more effective, safer or more cost
efficient than competing drugs, the possibility that our efforts to
acquire or in-license and develop additional drug candidates may fail,
our lack of sustained revenue history, our limited marketing experience,
our dependence on third parties for clinical trials, manufacturing,
distribution and quality control and other risks that are described in
further detail in the Company's reports filed with the Securities and
Exchange Commission. We do not plan to update any such
forward-looking statements and expressly disclaim any duty to update the
information contained in this press release except as required by law.
SPECTRUM PHARMACEUTICALS, INC.®, FUSILEV®, FOLOTYN®, ZEVALIN®,
MARQIBO®, and BELEODAQ® are registered trademarks of Spectrum
Pharmaceuticals, Inc. and its affiliates. REDEFINING CANCER CARE™
and the Spectrum Pharmaceuticals logos are trademarks owned by Spectrum
Pharmaceuticals, Inc. Any other trademarks are the property of
their respective owners.
© 2014 Spectrum Pharmaceuticals, Inc. All Rights Reserved
Vice President, Strategic
Planning & Investor Relations
Source: Spectrum Pharmaceuticals
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