US RESIDENTS: Please note that the information contained in this page is not intended for US residents. Please visit our US webpage. www.actelion.us

OUR PAH FRANCHISE

Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs. Actelion’s PAH franchise encompasses oral, inhaled and intravenous formulations of compounds, for patients at various stages in the course of this disease (WHO Functional Classes II–IV), enabling us to deliver treatments across the entire continuum of care.

OPSUMIT®

OPSUMIT®

Opsumit(macitentan), an orally available endothelin receptor antagonist, resulted from a tailored drug discovery process by Actelion.

Opsumit is commercially available in over 45 markets, including the US (since November 2013), Germany (since January 2014) and Japan (since June 2015). The registration process for other countries is ongoing.

TRACLEER®

TRACLEER®

Tracleer (bosentan), an orally available endothelin receptor antagonist, was the first oral treatment approved for PAH.
Tracleer is commercially available in over 60 markets, including the US (since November 2001), the EU (since May 2002) and Japan (since April 2005).

In addition to the PAH indication, Tracleer is approved in the EU for reducing the number of new digital ulcers in patients suffering from systemic sclerosis and ongoing digital ulcer disease.

UPTRAVI®

UPTRAVI®

Uptravi (selexipag), originally discovered and synthesized by Nippon Shinyaku, is the only approved oral, selective IP receptor agonist targeting the prostacyclin pathway in PAH.

Uptravi is commercially available in 9 markets, including the US (since January 2016) and Germany (since June 2016) with marketing authorization granted in Australia, Canada, the EU, Japan, New Zealand, South Korea, Switzerland and the US. The registration process for other countries is ongoing.

VELETRI®

VELETRI®

Veletri (epoprostenol for injection), an intravenous prostacyclin, is stable at room temperature (77°F/25°C) for up to 24 hours, removing the need for patients to carry ice packs.

Veletri is commercially available in 17 markets, including the US since 2010, Switzerland and Canada (marketed as Caripul®) since 2012, Japan (marketed as Epoprostenol “ACT”) since 2013, and some European countries since 2013. The registration process for other countries is ongoing.

VENTAVIS®

VENTAVIS®

Ventavis (iloprost), an inhaled formulation of iloprost, is a synthetic compound structurally similar to prostacyclin (PGI2).

Actelion has marketed Ventavis in the US since 2007. Elsewhere it is marketed by Bayer Healthcare.

OUR SPECIALTY PRODUCTS

Actelion has specialty products alongside PAH in new therapeutic areas.

VALCHLOR®

VALCHLOR®

Valchlor (mechlorethamine) 0.016% gel is applied topically once daily to affected areas of the skin. Valchlor is currently only available in the US and is approved for the topical treatment of Stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy.

Valchlor is commercially available in the US (since 2013) and in Israel. In France, patients benefit from the drug under a temporary authorization for use ("ATU") program initiated during the second half of 2014.

In March 2017, the European Commission has granted marketing authorization for the use of Ledaga® (chlormethine gel) 160 micrograms/g for the treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL). Subject to fulfilling the agreed commitments and achieving market access in various countries, a potential first European launch of Ledaga is not expected before H1 2018.

ZAVESCA® (MIGLUSTAT)

ZAVESCA® (MIGLUSTAT)

Zavesca, an orally active inhibitor of glucosylceramide synthase, is used as monotherapy in adult patients with mild to moderate type I Gaucher disease (GD1) for whom enzyme replacement therapy is not a therapeutic option. Zavesca is commercially available for the treatment of GD1 in 47 markets, including the US and the EU (since 2003).

In the EU, Zavesca is also indicated for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C (NP-C) disease, a rare neurodegenerative genetic disorder. Zavesca is commercially available for the treatment of
NP-C in 46 markets, including the EU (since 2009) and Japan (since 2012, marketed as Brazaves®).

"I enjoy working for Actelion because ultimately we help sick patients."

Regional Manager, Sales and Marketing, Brazil