News

Opsynvi, Macitentan Plus Tadalafil, Approved in Canada for PAH

Opsynvi, a fixed-dose oral combination of macitentan and tadalafil, was approved by Health Canada for the long-term treatment of pulmonary arterial hypertension (PAH). It is for use by patients on stable doses of its two active compounds: macitentan (sold as Opsumit) at 10 mg and tadalafil (sold as Adcirca) at 40 mg. Marketed…

Genetic Analysis Spots Cancer Therapies With Potential to Treat PH

By analyzing how medications affect the genetic activity of cells, researchers developed a computational pipeline that may be useful in repurposing cancer treatments for use in other diseases. Leveraging this pipeline, the team identified two compounds, I-BET762 and BRD2889, that might be refined — more quickly and at lower cost…

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

FDA Declines to Approve Tyvaso DPI, Citing Inspection Issue

Due to problems at a third-party facility, the U.S. Food and Drug Administration (FDA) has decided to not approve Tyvaso DPI, a dry powder inhaled formulation of treprostinil, at this time. According to United Therapeutics, the medication’s developer, the issue is expected to be dealt with soon. The company anticipates…

Oxygen Therapy May Ease Lung Pressure in CTEPH Before Angioplasty

Oxygen therapy may help relieve lung pressure in patients with chronic thromboembolic pulmonary hypertension (CTEPH) who are about to undergo balloon pulmonary angioplasty, a procedure in which balloons are used to open blood vessels that have been chronically narrowed or blocked, according to a new study. Researchers found a decrease…

PH Patients at High Risk of Misusing Opioids After Surgery

People with pulmonary hypertension (PH) who start treatment with opioids to manage pain after undergoing surgery are at high risk of continuing to take these addictive medications for longer than required, new research suggests. Indeed, hypertension was one of eight specifically identified “top risk factors” for “new persistent post-surgical…

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

Blocking Specific PI3K Protein Prevents PH in Animal Models

Targeting a protein called p110a, a part of the PI3K family of enzymes, may be useful for preventing or even reversing pulmonary hypertension, research done in cells and rodent models showed. “Targeted inhibition of [p110a] offers a disease-modifying treatment approach, which is readily accessible by small molecule inhibitors and…

Potential PAH Therapy, KER-012 Also Prevents Bone Loss in Rat Model

A lab version of an investigational treatment for bone disorders and pulmonary arterial hypertension (PAH), KER-012 prevented bone loss in a PAH rat model, its developer, Keros Therapeutics, reported. Separate preclinical data in a PAH rat model, presented by the company earlier this year, also demonstrated that KER-012 worked to…

Long Noncoding RNAs May Have Role in PH Onset, Rat Study Finds

Seven long noncoding RNA molecules were found to be associated with the development of pulmonary hypertension (PH) in an early study in rats, scientists reported. The researchers noted that some of these RNAs are known to regulate immune and inflammatory response pathways, which may participate in PH. “Our study…