Product

Mode of Delivery

Indication

Current Status
STUDY NAME CAPS

Target FDA
Approval Date

Our Territory

Target U.S.
Patient
Population Size

RemoSynch™
(Implantable System for Remodulin®)

Continuous intravenous via implantable pump

WHO Group 1 pulmonary arterial hypertension (PAH)

NDA to be resubmitted early 2017 following expected FDA complete response letter, following PMA resubmission (anticipated Oct 2016).

2017

United States, United Kingdom, Canada, France, Germany, Italy and Japan

6,000

RemUnity™
(treprostinil)

Continuous subcutaneous via pre-filled, semi-disposable pump

PAH

Pre-NDA

2018

Worldwide

6,000

Dinutiximab

Injection or infusion

Multiple GD2 expressing cancers

Phase II/III

2019-2023 for accel. approval and other reg. pathways

Worldwide

12,000

RemoPro™
(pain-free subcutaneous Remodulin® prodrug)

Continuous subcutaneous

PAH

Pre-clinical

2019

Worldwide

9,000

OreniPlus™
(Orenitram® in combination with approved background therapy)

Oral

PAH
(decrease morbitity and mortality)

Phase IV
FREEDOM-EV

2019

Worldwide

15,000

Ex-Vivo Lung Perfusion

Pre-transplant service providing extended preservation and assessment of donor lungs

End-stage lung disease

Phase II

2019

United States

8,000

Tysuberaprost™
(esuberaprost in combination with Tyvaso®)

Oral (esuberaprost)
Inhaled (Tyvaso)

PAH
(decrease morbidity and mortality)

Phase III
BEAT

2019

North America, Europe, Mexico, South America, Egypt, India, South Africa and Australia

2019

Tyvaso-ILD™
(treprostinil)

Inhaled

Pulmonary hypertension associated with idiopathic pulmonary fibrosis (WHO Group 3)

Phase III
INCREASE

2020

Worldwide

27,500

Aurora-GT™
(eNOS gene therapy)

Intravenous injection

PAH

Phase II/III
SAPPHIRE

2020

United States

10,000

RemoLiv™
(treprostinil)

Continuous intravenous

Liver transplant tolerance

Phase III
STARZL

2020

Worldwide

5,000

OreniLeft™
(treprostinil)

Oral

Pulmonary hypertension associated with left ventricular diastolic dysfunction
(WHO Group 2)

Phase III
SOUTHPAW

2021

Worldwide

50,000

Unexisome™
(exosome stem cell)

Infusion

Bronchopulmonary dysplasia

Pre-clinical

2022

Worldwide

12,000

OreniCell™
(treprostinil)

Oral

Reduce morbidity and mortality in patients with pulmonary hypertension associated with sickle cell disease
(WHO Group 5)

Phase II/III
IRONS

2022

Wordlwide

25,000

Manufactured Organs

Transplant

End-stage organ failure

Pre-Clinical

2023

Worldwide

> 30,000