Ceptaris Receives FDA Approval for VALCHLOR™ (mechlorethamine) Gel for the
Treatment of Stage IA and IB Mycosis Fungoides-Type Cutaneous T-Cell
Lymphoma in Patients Who Have Received Prior Skin-Directed Therapy
First and only FDA-approved topical formulation of mechlorethamine (nitrogen
Patient support and assistance programs to be established for VALCHLOR
MALVERN, Pa. -- August 26, 2013
Ceptaris Therapeutics, Inc., a privately held, specialty pharmaceutical
company, announced today that the U.S. Food and Drug Administration (FDA) has
granted marketing approval for the orphan drug VALCHLOR™ (mechlorethamine) gel
for the topical treatment of stage IA and IB mycosis fungoides-type cutaneous
T-cell lymphoma (CTCL) in patients who have received prior skin-directed
therapy. VALCHLOR is the first and only FDA-approved topical formulation of
mechlorethamine, commonly known as nitrogen mustard. VALCHLOR is a gel that is
applied topically once a day and dries on the skin.
"This is good news for patients and the treatment community," said Youn H.
Kim, M.D., Joanne and Peter Haas Jr. Professor for Cutaneous Lymphoma
Research, Professor of Dermatology, and Director, Multidisciplinary Cutaneous
Lymphoma Clinic, Stanford University School of Medicine. "We now have the
confidence of a FDA-approved product backed by evidence from a well-controlled
clinical trial that demonstrated clinically meaningful responses in the
majority of patients with stage IA and IB mycosis fungoides-type cutaneous
T-cell lymphoma (CTCL) who have received prior skin-directed therapy."
Mechlorethamine is a chemotherapeutic agent previously approved for
intravenous treatment of mycosis fungoides, the most common type of CTCL.
Topical mechlorethamine preparations are currently recommended for the
treatment of early stage CTCL by the National Comprehensive Cancer Network
(NCCN). Prior to the approval of VALCHLOR, there were no FDA-approved topical
mechlorethamine products; only non-standardized, pharmacy-compounded petroleum
ointment or aqueous-based topical preparations were available. Information
about pharmacy-compounded preparations is not required to be submitted to or
reviewed by FDA prior to their use by patients. Therefore, such preparations
do not undergo the same rigorous FDA review as FDA-approved products.
The availability of VALCHLOR will allow physicians to treat patients with
stage IA and IB mycosis fungoides-type CTCL who have received prior
skin-directed therapy with a FDA-approved version of topical mechlorethamine.
In addition to consistent, controlled manufacturing processes, VALCHLOR will
be provided with labeling, which includes data and instructions for proper use
to help achieve the best possible clinical results.
"The use of topical mechlorethamine has been documented over several decades,
but this is the first time that a product has gone through the rigorous FDA
approval process," said Stuart R. Lessin, M.D., former Director of Dermatology
at the Fox Chase Cancer Center, President of the Board of Directors of the
Cutaneous Lymphoma Foundation and lead investigator in the VALCHLOR pivotal
trial. "Not only is VALCHLOR manufactured under FDA’s good manufacturing
practices, but it will also be accompanied by patient support and assistance
programs which are not currently available with compounded mechlorethamine."
Results of the VALCHLOR Pivotal Trial
The approval of VALCHLOR was based on a randomized, observer-blinded,
non-inferiority pivotal trial comparing VALCHLOR to a pharmacy-compounded
mechlorethamine preparation in patients with stage IA-IIA MF-type CTCL.
Patients had received at least one prior skin-directed therapy. Qualifying
prior therapies included topical corticosteroids, phototherapy, Targretin®
gel, and topical nitrogen mustard (mechlorethamine). Patients were not
required to be refractory to or intolerant of prior therapies. In the thirteen
center study, 260 patients (the vast majority of whom were IA and IB) were
enrolled (1:1 randomization), making it the largest randomized study ever
conducted in mycosis fungoides-type CTCL. Results of the study were published
earlier this year in JAMA Dermatology.^1
In the study, 60% of patients treated with VALCHLOR had a confirmed response
at 12 months, defined as reduction of at least 50% in the Composite Assessment
of Index Lesion Severity (CAILS) score, while 48% of those treated with the
compounded control achieved a confirmed response. Complete responses
constituted a minority of the CAILS overall response. CAILS responses were
seen as early as 1 month, with further responses observed through 11 months of
treatment. No systemic absorption of mechlorethamine was detected with
Important Safety Information for VALCHLOR
VALCHLOR is for topical dermatologic use only. VALCHLOR is a cytotoxic drug.
Avoid direct skin contact with VALCHLOR in individuals other than the patients
due to risk of dermatitis, mucosal injury and secondary cancers. The use of
VALCHLOR is contraindicated in patients with a history of severe or systemic
hypersensitivity to mechlorethamine or inactive ingredients. Contact with
mucous membranes, especially those of the eyes, must be avoided. Exposure of
the eyes to mechlorethamine may cause pain, burns, inflammation, photophobia,
blurred vision and in some cases severe and long-lasting injury to the eye.
Patients should be monitored for non-melanoma skin cancers during and after
treatment with VALCHLOR. The most common adverse reaction to VALCHLOR is
dermatitis, which in some cases may be severe and require dosing changes or
discontinuation. Elderly patients may be more susceptible to dermatitis. Women
should avoid becoming pregnant or nursing while using VALCHLOR due to
potential hazard to the fetus. VALCHLOR is an alcohol-based gel. Avoid fire,
flame and smoking until the gel has dried.
Commercialization of VALCHLOR
VALCHLOR will be distributed by Accredo Specialty Pharmacy with a target
availability in the 4^th quarter of 2013. Physicians will be able to prescribe
VALCHLOR by visiting www.valchlor.com. Patient support and assistance programs
will be available to patients. The program services offered will include
reimbursement and financial support for eligible patients, as well as disease
and product information.
On July 30, 2013, Ceptaris signed an agreement to merge with Actelion US
Holding Company, a subsidiary of Actelion Ltd. (SIX: ATLN). The approval of
VALCHLOR is a condition of closing the merger. Both companies anticipate that
Actelion's expertise in rare diseases will facilitate the delivery of VALCHLOR
"The FDA approval of VALCHLOR is an important milestone which was achieved
through the dedicated efforts of our team, our clinical investigators, and the
patients who participated in our clinical studies," said Stephen Tullman,
President and Chief Executive Officer, Ceptaris Therapeutics, Inc. "With this
approval, we look forward to working with Actelion to close the merger and
make VALCHLOR available to patients."
About Mycosis Fungoides and Cutaneous T-Cell Lymphoma
Mycosis fungoides is the most common type of cutaneous T-cell lymphoma, a rare
form of non-Hodgkin's lymphoma. The cause of mycosis fungoides remains unknown
and there is no known cure. Unlike most non-Hodgkin's lymphomas, mycosis
fungoides is caused by malignant T-cells. The malignant T-cells in the body
initially migrate to the skin, causing lesions to appear. These lesions
typically begin as what appears to be a rash and may progress to form plaques
and disfiguring tumors. Early stage cases may be confused with other skin
conditions until a definitive diagnosis is made based upon skin biopsy. Most
cases of mycosis fungoides are early stage and are diagnosed in patients over
the age of 50.
About Ceptaris Therapeutics
Ceptaris Therapeutics, Inc. is a privately held, specialty pharmaceutical
company that has developed a proprietary gel formulation of mechlorethamine
hydrochloride for the treatment of stage IA and IB mycosis fungoides, a type
of CTCL. VALCHLOR (mechlorethamine) gel is the first and only FDA-approved
topical mechlorethamine product available to treat the signs and symptoms of
this rare cancer. Please visit http://www.ceptaris.com for more information.
This release includes forward-looking statements concerning the Company
including expectations regarding regulatory filings. The statements are based
on assumptions about many important factors, including the following, which
could cause actual results to differ materially from those in the
forward-looking statements: satisfaction of regulatory and other requirements;
actions of regulatory bodies and other governmental authorities; changes in
laws and regulations; product quality or patient safety issues. The
information contained in this press release was accurate at the time of
issuance and Ceptaris assumes no responsibility for updating the information
to reflect subsequent developments.
^1 Lessin SR, Duvic M, Guitart J, Pandya AG, Strober BE, Olsen EA, et al.
Topical chemotherapy in cutaneous T-cell lymphoma: Positive results of a
randomized, controlled, multicenter trial testing the efficacy and safety of a
novel mechlorethamine, 0.02%, gel in mycosis fungoides. JAMA Dermatol.
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