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2016-12-16

Actelion Pharmaceuticals Ltd: Actelion receives positive CHMP opinion for chlormethine gel (Ledaga) for the treatment of MF-CTCL

Actelion Pharmaceuticals Ltd / Actelion receives positive CHMP opinion for
chlormethine gel (Ledaga) for thetreatment of MF-CTCL . Processed and
transmitted by Nasdaq Corporate Solutions.The issuer is solely responsible
for the content of this announcement.
ALLSCHWIL/BASEL, SWITZERLAND - 16 December 2016
-
Actelion Ltd (SIX: ATLN) announced today that the Committee for Medicinal
Products for Human Use (CHMP), the scientific committee of the European
Medicines Agency (EMA), issued a positive opinion for the use of chlormethine
gel 160 micrograms/g (Ledaga®) for the treatment of mycosis fungoides-type
cutaneous T-cell lymphoma (MF-CTCL) in adult patients and recommended that
the European Commission approves the product.

The CHMP opinion is based on the results of the pivotal 201 study, the largest
randomized controlled study ever conducted in MF-CTCL involving 260 patients.
In this study 77% of patients who were treated for at least 6 months with
chlormethine gel achieved a clinical response, in the Composite Assessment of
Index Lesion Severity (CAILS) score, while 59% of those treated with the
compounded control had a clinical response. A response was defined as an at
least 50% improvement in the baseline CAILS score. Complete response was
achieved in 19% of patients versus 15% of patients treated with the
compounded control. Reductions in mean lesion severity were seen as early as
four weeks into the study, with further reductions observed with continuing
therapy. The time to first confirmed response favored chlormethine gel
(Ledaga) compared to the compounded control.

MF-CTCL is a rare, potentially life-threatening immune system cancer that
appears in the skin. MF-CTCL is usually a chronic disease and the course of
disease in individual patients is unpredictable. In around 10% of cases,
MF-CTCL cells can metastasize to other body tissues, including the liver,
spleen and lungs.

In the 201 study, the most frequent adverse reactions reported with
chlormethine gel were skin related: dermatitis (54.7%; e.g., skin irritation,
erythema, rash, urticaria, skin-burning sensation, pain of the skin),
pruritus (20.3%), skin infections (11.7%), skin ulceration and blistering
(6.3%), and skin hyperpigmentation (5.5%). No systemic absorption of
chlormethine was detected with treatment.

A CHMP positive opinion is one of the final steps before marketing
authorization is granted by the European Commission. The European Commission
is expected to issue a final decision by the end of February 2017.

Actelion has agreed to a list of recommendations from the CHMP
(post-authorization measures) with regards to release of the product in
Europe. Subject to the agreed recommendations and achieving market access in
different countries, a potential first launch of Ledaga could occur at the
end of 2017 at the earliest.

ABOUT CHLORMETHINE GEL (LEDAGA)

Chlormethine is an alkylating drug indicated for the treatment of mycosis
fungoides-type cutaneous T-Cell lymphoma (MF-CTCL) formulated as a topical,
once-daily, colorless gel (Ledaga).

Chlormethine gel, under the brand name Valchlor® (mechlorethamine) is
commercially available in the US (since 2013) and in Israel through special
import authorization procedure (since 2016).

In France, patients benefit from the drug under a temporary authorization for
use ("ATU") program initiated during the second half of 2014.

###

Notes to Editor:

ABOUT MF-CTCL

Mycosis fungoides-type cutaneous T-Cell lymphoma (MF-CTCL) is a rare, but
serious and life-threatening, immune system cancer that appears in the skin.
MF-CTCL is the most common form of cutaneous T-cell lymphoma.

MF-CTLC typically appears in patients over 50 years of age (median age is 54),
and is more common in men. It presents first as dry skin and a red rash, with
or without itching. As a result, MF-CTLC is often mistaken for eczema or
psoriasis, delaying diagnosis.

MF-CTLC goes on to form scaly plaques on the skin, which can cover small or
large areas of the skin. Large bumps or tumor nodules may also develop, and
lymph nodes may be involved.

While MF-CTCL is usually a chronic disease, the course of disease in
individual patients is unpredictable with some patient progressing into
advanced stages. In around 10% of cases, MF-CTCL cells can metastasize into
other body tissues, including the liver, spleen and lungs.

Current research suggests that patients who are diagnosed in early stages of
MF-CTCL have a normal life expectancy, however the average time to diagnosis
ranges from two to seven years. An important therapeutic objective in
treating MF-CTLC is prevention of disease progression. Failure to maintain
MF-CTLC in its early stages results in a drastically reduced median survival.

ABOUT STUDY 201

Study 201 was a multicenter, randomized, observer-blinded, active-controlled,
12-month study of Stage I and IIA MF-type CTCL patients, conducted in 13
centers in the US to evaluate the efficacy and safety of chlormethine gel
compared with chlormethine HCl 0.02% compounded in Aquaphor®ointment. In
total, 260 patients were randomized 1:1 to topical treatment with
chlormethine gel or chlormethine HCl 0.02% compounded in Aquaphor®ointment
once daily for up to 12 months.

In the study, 77% of patients treated with chlormethine gel had a clinical
response at 12 months, in the Composite Assessment of Index Lesion Severity
(CAILS*) score, while 59% of those treated with the compounded control
achieved a confirmed response. (*A response was defined as an at least 50%
improvement in the baseline CAILS score).

Complete response was achieved in 19% of patients versus 15% of patients
treated with the compounded control. Reductions in mean lesion severity
(CAILS) were seen as early as four weeks, with further reductions observed
with continuing therapy. The time to first confirmed response favored
chlormethine gel (Ledaga) compared to the compounded control.

The most frequent adverse reactions reported with chlormethine gel were skin
related: dermatitis (54.7%; e.g., skin irritation, erythema, rash, urticaria,
skin-burning sensation, pain of the skin), pruritus (20.3%), skin infections
(11.7%), skin ulceration and blistering (6.3%), and skin hyperpigmentation
(5.5%). No systemic absorption of chlormethine was detected with treatment.

Actelion Ltd.

Actelion Ltd. is a leading biopharmaceutical company focused on the discovery,
development and commercialization of innovative drugs for diseases with
significant unmet medical needs.

Actelion is a leader in the field of pulmonary arterial hypertension (PAH).
Our portfolio of PAH treatments covers the spectrum of disease, from WHO
Functional Class (FC) II through to FC IV, with oral, inhaled and intravenous
medications. Although not available in all countries, Actelion has treatments
approved by health authorities for a number of specialist diseases including
Type 1 Gaucher disease, Niemann-Pick type C disease, Digital Ulcers in
patients suffering from systemic sclerosis, and mycosis fungoides type
cutaneous T-cell lymphoma.

Founded in late 1997, with now over 2,500 dedicated professionals covering all
key markets around the world including Europe, the US, Japan, China, Russia
and Mexico, Actelion has its corporate headquarters in Allschwil / Basel,
Switzerland.

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®). All
trademarks are legally protected by their respective owners.

For further information please contact:

Andrew C. Weiss
Senior Vice President, Head of Investor Relations&Corporate Communications
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
+41 61 565 62 62
www.actelion.com

The above information contains certain "forward-looking statements", relating
to the company's business, which can be identified by the use of
forward-looking terminology such as "estimates", "believes", "expects",
"may", "are expected to", "will", "will continue", "should", "would be",
"seeks", "pending" or "anticipates" or similar expressions, or by
discussions of strategy, plans or intentions. Such statements include
descriptions of the company's investment and research and development
programs and anticipated expenditures in connection therewith, descriptions
of new products expected to be introduced by the company and anticipated
customer demand for such products and products in the company's existing
portfolio. Such statements reflect the current views of the company with
respect to future events and are subject to certain risks, uncertainties and
assumptions. Many factors could cause the actual results, performance or
achievements of the company to be materially different from any future
results, performances or achievements that may be expressed or implied by
such forward-looking statements. Should one or more of these risks or
uncertainties materialize, or should underlying assumptions prove incorrect,
actual results may vary materially from those described herein as
anticipated, believed, estimated or expected.

Press Release PDF
http://hugin.info/131801/R/2066035/775601.pdf

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This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Actelion Pharmaceuticals Ltd via Globenewswire

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