News

The U.S. Food and Drug Administration (FDA) has approved a new, third-generation inhalation device, the TD-300/A, for use with Tyvaso (treprostinil) Inhalation Solution, United Therapeutics Corporation announced. Tyvaso is a prostacyclin vasodilator indicated for the treatment of pulmonary arterial hypertension (PAH) to improve exercise ability. Tyvaso was…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

A heart chamber malfunction leads to worse survival in pulmonary arterial hypertension (PAH) patients who have had a double lung transplant, a study reports. The malfunction, known as diastolic dysfunction, occurs when the heart’s left ventricle is unable to fill up with blood before pumping it out to the body. Researchers…

Patients with pulmonary arterial hypertension (PAH) taking Arena Pharmaceuticals‘ next-generation therapy Ralinepag (APD811) showed significant improvements in blood flow and exercise capacity in a Phase 2 clinical trial. Results of the study (NCT02279160) will be presented at the American College…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

Reviva Pharmaceuticals has had a meeting with the U.S. Food and Drug Administration that paves the way for a Phase 2 clinical trial of its neurodegenerative therapy RP5063 in pulmonary arterial hypertension. The meeting dealt with an investigational new drug application the company will submit on RP5063. What is known in therapy development circles…

Professor David Kiely, MD, and his team at Sheffield Teaching Hospitals NHS Foundation Trust in the United Kingdom, received a national award from the National Institute for Health Research (NIHR) Clinical Research Network and Royal College of Physicians. Kiely, who is director of the Sheffield Pulmonary Vascular Disease Unit…

Patients with a disease that causes abnormal blood vessel formation in skin, mucous membranes and some organs are at risk of developing different types of pulmonary hypertension, a study shows. The organs affected by the blood-vessel condition, hereditary hemorrhagic telangiectasia (HHT), include the lungs, liver and brain. The types of pulmonary…

The European Patent Office has issued a notice of allowance to the biopharmaceutical company SteadyMed for its enhanced infusion-site pain reduction technology called the PatchPump infusion system. A notice of allowance is a document sent to a patent applicant indicating that a patent examiner has issued the requested patent. SteadyMed’s…

Actelion’s Opsumit (macitentan) made it easier for blood to pass through the lung blood vessels of chronic thromboembolic pulmonary hypertension (CTEPH) patients, a Phase 2 clinical trial suggests. In addition to lowering patients’ lung blood pressure, Opsumit also improved their ability to exercise, and was safe, researchers said. The trail…

IV delivery of stem cells to rats with pulmonary arterial hypertension (PAH) reduced the animals’ harmful lung blood vessel remodeling and improved their blood flow, a study reports. Researchers said the implication was that the stem cells could be used as a treatment for PAH. They used a type of stem…

A top pulmonary hypertension expert will be among more than 80 speakers at this month’s Rare Disease & Orphan Products Breakthrough Summit in Washington. The two-day event Oct. 16-17, which is in its sixth year, will be hosted by the nonprofit National Organization for Rare Disorders (NORD). It…