The U.S. Food and Drug Administration (FDA) has approved Yutrepia (treprostinil) inhalation powder for adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease.
PAH
PAH is a rare condition affecting the heart and lungs. It is characterized by abnormally high blood pressure in the pulmonary artery. Symptoms include shortness of breath during exercise and fainting spells. The symptoms tend to get worse over time and may include dizziness, swelling of the ankles or legs, chest pain, and a racing pulse. Some cases of PAH are due to genetic changes in the BMPR2 gene. PAH can also occur secondary to underlying conditions such as connective tissue diseases, HIV infection, chronic hemolytic anemia, and congenital heart disease. PAH can also be induced by certain drugs and toxins.
Treprostinil
Treprostinil is a prostacyclin analog inhalation powder designed to enhance deep-lung delivery. The approval follows positive results from the phase 3 INSPIRE trial (NCT03399604) which was an open-label, multicenter study of treprostinil in patients with PAH who are naïve to inhaled treprostinil or who are transitioning from Tyvaso (nebulized treprostinil).
Patients receiving stable doses of nebulized treprostinil successfully transitioned to treprostinil inhalation powder with no significant safety concerns. Patients receiving treprostinil in addition to oral therapy also showed favorable tolerability. Almost all patients experienced treatment-related events consistent with the known side effects of inhaled treprostinil including cough, throat irritation, oropharyngeal pain, headache, dizziness, diarrhea, chest discomfort, nausea, dyspnea, and flushing. However, these adverse events were mild to moderate and did not affect patients’ ability to participate in the trial. The adverse event incidence rate was less in patients transitioning from nebulized treprostinil compared to those initiating use of treprostinil.
Treprostinil is currently being investigated further in the ASCENT clinical trial (NCT06129240), an open-label, prospective, multicenter study evaluating the safety and tolerability of dry powder inhaled treprostinil in patients with pulmonary hypertension associated with interstitial lung disease.
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To learn more about PAH and other rare lung conditions, visit https://checkrare.com/diseases/lung-diseases/