The FDA is expected to decide on the company’s application by Aug. 22, 2026. ・Molbreevi had previously been granted FDA’s fast track, breakthrough therapy and orphan drug designations. ・The company ...

The FDA granted orphan drug designation to deupirfenidone, also known as LYT-100, for treating patients with idiopathic pulmonary fibrosis, according to a press release from PureTech Health.The ...

Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the 13 hot stocks to buy with the highest upside potential. As of February 13, ...

One challenge is limited commercial synergy. Rare disease markets are often fragmented, with small prescriber bases and ...

Dyne Therapeutics (NASDAQ:DYN) is one of the 17 biotechnology stocks with more than 50% upside. As of the February 12 closing ...

Rilzabrutinib is an investigational, oral reversible Bruton tyrosine kinase inhibitor. The Food and Drug Administration (FDA) has granted Orphan Drug designation to rilzabrutinib for the treatment of ...

In this episode of Ask the Expert, Susan J. Schniepp, Nelson Labs, and Siegfried Schmitt, Parexel, discuss the benefits of orphan drug development and how a mid-sized company can work with regulators ...

SHELTON, CONNECTICUT / ACCESS Newswire / February 12, 2026 / NanoViricides, Inc., a publicly traded company (NYSE ...

DUBLIN--(BUSINESS WIRE)--The "Orphan Drugs - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering. The global market for Orphan Drugs was estimated at US$179.5 ...

Aug. 1, 2005 — Europe has approved orphan drugs pegylated arginine deiminase for the treatment of hepatocellular carcinoma, soluble yeast beta-1,3/1,6-glucan for the prophylaxis of mucositis due to ...

April 11, 2006 — The US Food and Drug Administration (FDA) has approved orphan drug status for CEP-701 in the treatment of acute myeloid leukemia; the monoclonal antibody ChAglyCD3 for new-onset type ...