Actelion launches VALCHLOR (mechlorethamine) gel 0.016% in the US

  Actelion launches VALCHLOR (mechlorethamine) gel 0.016% in the US

  *First and only FDA-approved topical formulation of mechlorethamine
    available for patients with stage IA and IB mycosis fungoides-type
    cutaneous T-cell lymphoma who have received prior skin-directed therapy
  *Actelion establishes a patient assistance and support program

Business Wire

SOUTH SAN FRANCISCO, Calif. -- November 18, 2013

Actelion (SIX: ATLN) announced today that VALCHLOR™ (mechlorethamine), the
first and only FDA-approved topical formulation of mechlorethamine, is now
available for patients in the United States (US). VALCHLOR, a gel which is
applied topically once a day, is an alkylating drug indicated to treat
patients with stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma
(MF-CTCL) who have received prior skin-directed therapy. VALCHLOR is an orphan
drug that was acquired by Actelion US Holding Company, a subsidiary of
Actelion Ltd., on September 18, 2013 as part of a merger with Ceptaris
Therapeutics, Inc. VALCHLOR is distributed in the US by Accredo Specialty

"The availability of VALCHLOR is exciting news for patients and the treatment
community. Physicians now have the option to treat appropriate MF-CTCL
patients with the first formulation of topical mechlorethamine that is
FDA-approved based on rigorous clinical evidence to support its use,” 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.

"The use of topical mechlorethamine has been documented over several decades,”
said Stuart 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. “With the
launch of VALCHLOR, physicians can prescribe with confidence knowing that an
FDA-approved formulation is now available, along with support and
financial-assistance programs for eligible patients to help facilitate
education and access."

Mycosis fungoides is the most common type of cutaneous T-cell lymphoma, a rare
form of non-Hodgkin's lymphoma. In the US, approximately 20,000 patients are
currently diagnosed with stage IA-IB MF-CTCL, qualifying it as a rare or
orphan disease.

“We look forward to partnering with dermatology and oncology specialists to
bring this treatment to appropriate patients with MF-CTCL,” said Bill Fairey,
president of Actelion Pharmaceuticals US Inc. “Actelion has deep expertise in
rare, orphan diseases which we are utilizing to help ensure VALCHLOR is
accessible to patients who may benefit from this important therapy.”


Actelion has established VALCHLOR Support™, an assistance program to help
eligible patients successfully start and remain on VALCHLOR therapy. The
program, administered by Accredo Specialty Pharmacy (,
includes reimbursement and financial support for eligible patients, as well as
disease and product information.

“MF-CTCL can have a significant impact on the appearance and daily lives of
patients affected by the disease,” said Susan Thornton, chief executive
officer of the Cutaneous Lymphoma Foundation. “We are thrilled to have a new
treatment option in VALCHLOR, supported by an assistance program for eligible
patients to help ensure access.”

For more information on the VALCHLOR Support program, patients can call
1-855-4-VALCHLOR (1-855-482-5245) between 9:00am – 11:00pm Eastern Time, or


VALCHLOR (mechlorethamine) gel 0.016% is an alkylating drug indicated for the
topical treatment of stage IA and IB mycosis fungoides-type cutaneous T-cell
lymphoma (MF-CTCL) in patients who have received prior skin-directed therapy.
VALCHLOR is a gel which is applied topically once a day. Mechlorethamine,
commonly known as nitrogen mustard, is a chemotherapeutic agent previously
approved for intravenous treatment of mycosis fungoides, the most common type
of cutaneous T-Cell lymphoma. Mechlorethamine is one of the suggested
skin-directed treatment regimens for early-stage MF-CTCL according to the
latest National Comprehensive Cancer Network® (NCCN®) Clinical Practice
Guidelines in Oncology (NCCN Guidelines®)^1. VALCHLOR is the first and only
FDA-approved topical formulation of mechlorethamine.

For more information and full prescribing information visit


Caregivers must wear disposable nitrile gloves during application and avoid
direct skin contact. 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

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.


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.


Actelion Ltd is a biopharmaceutical company with its corporate headquarters in
Allschwil/Basel, Switzerland. Actelion's first drug Tracleer® (bosentan), an
orally available dual endothelin receptor antagonist, has been approved as a
therapy for pulmonary arterial hypertension. Actelion markets Tracleer through
its own subsidiaries in key markets worldwide, including the United States
(based in South San Francisco), the European Union, Japan, Canada, Australia
and Switzerland. Actelion, founded in late 1997, is a leading player in
innovative science related to the endothelium - the single layer of cells
separating every blood vessel from the blood stream. Actelion's over 2,300
employees focus on the discovery, development and marketing of innovative
drugs for significant unmet medical needs. Actelion shares are traded on the
SIX Swiss Exchange (ticker symbol: ATLN) as part of the Swiss blue-chip index
SMI (Swiss Market Index SMI®).


^1Referenced with permission from the NCCN Clinical Practice Guidelines in
Oncology (NCCN Guidelines®) for Non-Hodgkin's Lymphomas V.2.2013. © National
Comprehensive Cancer Network, Inc 2013. All rights reserved. Accessed
[November 14, 2013]. To view the most recent and complete version of the
NCCN®, NCCN GUIDELINES®, and all other NCCN Content are trademarks owned by
the National Comprehensive Cancer Network, Inc.

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Sam Brown Inc.
Mike Beyer, 773-463-4211
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