While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…
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A real-world, multi-center study in Poland confirms the effectiveness and safety of two-year treatment with Esbriet (pirfenidone) for people with idiopathic pulmonary fibrosis (IPF). The study “A multicentre retrospective observational study on Polish experience of pirfenidone therapy in patients with idiopathic pulmonary fibrosis: the PolExPIR study” was published…
Ofev (nintedanib), an oral medication by Boehringer Ingelheim, is now approved to treat people with progressive fibrosing interstitial lung diseases (ILDs) in the U.S. and Canada. These approvals, by the U.S. Food and Drug Administration (FDA) and Health Canada, come on the heels of findings from the…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Ofev, an approved treatment of idiopathic pulmonary fibrosis (IPF), may be considered as a monotherapy for patients who also have non‐small cell lung cancer and are unable to tolerate cancer chemotherapy, a case study reported. …
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
The U.S. Food and Drug Administration (FDA) has designated AMP945, Amplia Therapeutics’ investigational anti-fibrotic therapy for idiopathic pulmonary fibrosis (IPF), an orphan drug, supporting its development, the company announced. Its decision came about one month after AMP945 also received orphan drug status as a potential pancreatic cancer…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
Repeat hospitalizations for respiratory problems in people with chronic idiopathic interstitial pneumonia (c-IIP) — a group of chronic lung diseases that also include idiopathic pulmonary fibrosis (IPF) — are associated with poor long-term survival, a study reported. The study, “Characteristics of patients with chronic idiopathic interstitial…
The French Medicines Agency has authorized Genkyotex to launch a Phase 1 trial to evaluate the safety and pharmacological properties of high-dose setanaxib, the company’s investigational oral treatment for idiopathic pulmonary fibrosis (IPF). Setanaxib (formerly known as GKT831) is an investigational anti-fibrotic therapy that is thought…
