News

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…

An inherited virus’ shift from a dormant to an active mode appears to be linked to the development of pulmonary arterial hypertension in rats, a study reports. Scientists may be able to develop PAH therapies around the idea of preventing the human endogenous retrovirus K (HERV-K) from becoming active, the study added.

Pulmonary artery dilation speeds up the progression of pediatric idiopathic and heritable pulmonary arterial hypertension (PAH), a Japanese study shows. Dilation of the main pulmonary artery (MPA) and narrowing of the peripheral pulmonary artery are both characteristic of PAH. Previous studies have shown that MPA dilation is linked to higher pulmonary artery…

A University of Oregon researcher played a key role in the early work that led to Complexa’s pulmonary hypertension therapy CXA-10 going to clinical trials. The treatment is designed to prevent inflammation and lung scarring. It will be investigated in two clinical trials as a non-steroid alternative for treating pulmonary…

Pulmonary arterial hypertension (PAH) impacts multiple aspects of patients’ and caregivers’ lifes, a new survey from China shows. The study, “The impact and financial burden of pulmonary arterial hypertension on patients and caregivers: results from a national survey,” was published in the journal Medicine. PAH is…