Treprostinil
- Generic Name
- Treprostinil
- Brand Names
- Orenitram, Remodulin, Tyvaso, Trepulmix
- Drug Type
- Small Molecule
- Chemical Formula
- C23H34O5
- CAS Number
- 81846-19-7
- Unique Ingredient Identifier
- RUM6K67ESG
- Background
Treprostinil is a stable tricyclic analogue of prostacyclin that promotes the vasodilation of pulmonary and systemic arterial vascular beds and the inhibition of platelet aggregation. It reduces symptoms in patients with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease. The first agent approved for the treatment of PAH was epoprostenol, a synthetic prostacyclin that significantly increases patients' quality of life. However, the use of epoprostenol is limited due to its short half-life (3-5 min) and instability at room temperature. The use of more stable alternatives such as treprostinil provides patients with PAH with more treatment options.
Treprostinil was approved by the FDA in 2002 for the treatment of pulmonary arterial hypertension. It is available in the following routes of administration: subcutaneous, intravenous, inhaled and oral. The first generic form of treprostinil became available in 2019.
- Indication
The FDA has indicated treprostinil for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease to improve exercise ability. It is also used to treat pulmonary arterial hypertension in patients requiring transition from epoprostenol. The Health Canada label specifies that treprostinil is indicated for the long-term treatment of pulmonary arterial hypertension in NYHA Class III and IV patients who did not respond adequately to conventional therapy.
L24244
- Associated Conditions
- Pulmonary Arterial Hypertension (PAH), Pulmonary Hypertension (PH)
Court Dismisses United Therapeutics' Challenge Against Liquidia's YUTREPIA, Clearing Path for May Launch
• U.S. District Court Judge Kelly dismissed United Therapeutics' cross-claim challenging Liquidia's NDA amendment for YUTREPIA, ruling that UTHR failed to establish standing.
• The FDA can now grant final approval for YUTREPIA (treprostinil) inhalation powder for both PAH and PH-ILD indications after regulatory exclusivity expires on May 23, 2025.
• Liquidia remains on track for a potential commercial launch with a PDUFA goal date of May 24, 2025, positioning YUTREPIA to potentially become a first-choice prostacyclin therapy for pulmonary hypertension patients.
Liquidia Secures $100M Financing to Advance Yutrepia for Pulmonary Hypertension Treatment
• Liquidia Corporation has amended its agreement with Healthcare Royalty to potentially add up to $100 million in financing, with an initial $25 million available at closing.
• The funds will support Yutrepia's commercial development for pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), along with clinical trials for pediatric patients.
• Additional funding tranches include $50 million upon first commercial sale following FDA approval and $25 million if Yutrepia achieves over $100 million in net sales by June 2026.
Pipeline for Pulmonary Arterial Hypertension Treatment Expands with 55+ Companies Developing Novel Therapies
• DelveInsight's latest report reveals over 55 companies actively developing 55+ pipeline drugs for Pulmonary Arterial Hypertension treatment, indicating robust research activity in this therapeutic area.
• Breakthrough therapies like Sotatercept by Acceleron Pharma and LIQ861 by Liquidia Technologies are advancing through clinical trials, offering new hope for PAH patients.
• Multiple clinical trials are underway in early 2025, including studies by Actelion, Keros Therapeutics, Insmed Incorporated, and Merck Sharp & Dohme, focusing on novel treatment approaches.
United Therapeutics Completes Enrollment in Phase 3 TETON 1 IPF Trial of Inhaled Treprostinil
• United Therapeutics has fully enrolled its Phase 3 TETON 1 study, evaluating nebulized Tyvaso (treprostinil) for idiopathic pulmonary fibrosis (IPF).
• The TETON 1 trial, with 598 patients across the U.S. and Canada, is part of a larger program assessing inhaled treprostinil in IPF and progressive pulmonary fibrosis (PPF).
• Top-line data from the TETON 1 study is anticipated in the first half of 2026, potentially leading to a new treatment option for IPF patients.
• If successful, United Therapeutics plans to seek FDA approval to expand Tyvaso's label to include IPF, and pursue European marketing authorization.
Insmed's TPIP and Brensocatib Programs Show Promise in Rare Pulmonary Diseases
Leerink Partners maintains a Buy rating on Insmed with an $85 price target, highlighting significant potential in pulmonary arterial hypertension treatment with TPIP and brensocatib for non-cystic fibrosis bronchiectasis. The company's strategic position in addressing unmet needs in rare pulmonary diseases has sparked acquisition speculation.
Novel Pipeline Therapies Show Promise for Pulmonary Sarcoidosis Treatment
DelveInsight's latest pipeline report reveals over 10 companies actively developing treatments for pulmonary sarcoidosis. Key players including Novartis and aTyr Pharma are advancing innovative therapies, with CMK389 and ATYR1923 showing particular promise in clinical development.
Insmed's Brensocatib NDA Accepted by FDA with Priority Review; Anticipated Launch in Q3 2025
• The FDA has accepted Insmed's New Drug Application for brensocatib in bronchiectasis, granting Priority Review with a PDUFA target action date of August 12, 2025.
• Insmed anticipates launching brensocatib in the U.S. in the third quarter of 2025, pending FDA approval, with regulatory submissions planned for the EU, UK, and Japan in 2025.
• ARIKAYCE global revenue grew 19% in 2024, and Insmed projects 2025 revenues between $405 million and $425 million, representing double-digit growth.
• A Phase 3 study of TPIP in pulmonary hypertension associated with interstitial lung disease is planned for the second half of 2025.
Systemic Sclerosis Pipeline Shows Promise with 50+ Therapies in Development
• Over 50 active players are currently developing more than 50 pipeline therapies for the treatment of Systemic Sclerosis, indicating a robust and active research landscape.
• Key companies like Eicos Sciences, Kyowa Kirin, and Corbus Pharmaceuticals are advancing Systemic Sclerosis therapies, contributing to a competitive market.
• Promising therapies such as GS-248, KHK4827, and Paquinimod are in various stages of clinical development, offering potential new treatment options.
• Horizon Pharma initiated a Phase 2 clinical trial for HZN-825 BID, with an extension trial for participants completing the initial 52-week treatment period.
Supreme Court Declines to Review United Therapeutics' Patent, Clearing Path for YUTREPIA
• The U.S. Supreme Court rejected United Therapeutics' appeal regarding the invalidation of its U.S. Patent No. 10,716,793.
• This decision finalizes rulings that Liquidia's YUTREPIA does not infringe any valid claims of United Therapeutics' patents.
• Tentative FDA approval for YUTREPIA to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) was previously granted.
• Final FDA approval for YUTREPIA is anticipated after May 23, 2025, following the expiration of Tyvaso DPI's regulatory exclusivity.
United Therapeutics to Present Tyvaso DPI Data at CHEST 2024
• United Therapeutics will present data from the BREEZE open-label extension study of Tyvaso DPI, detailing long-term outcomes in pulmonary arterial hypertension patients.
• The company is sponsoring a symposium on Tyvaso DPI, focusing on clinical pearls and drug-device characteristics, at the CHEST 2024 Annual Meeting.
• Additional presentations will cover patient-reported outcomes, healthcare resource utilization, and the impact of inhaled treprostinil in pulmonary hypertension.
• United Therapeutics also sponsors the Women in Chest Medicine Annual Luncheon and the Advanced Practice Providers (APPs) in Chest Medicine Forum.
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