Takeda and Protagonist Therapeutics have submitted a new drug application to the FDA for rusfertide for adults with ...

Cellenkos® Inc., a clinical-stage biotechnology company developing allogeneic, off-the-shelf, regulatory T cell (Treg) ...

The US Food and Drug Administration has granted breakthrough therapy designation to Oculis’ neuroprotective candidate ...

As orphan drugs account for an increasing share of drugs approved, they are driving up the cost of drug launches and drug prices. In a new paper, America's Health Insurance Plans analyzes these rising ...

The Orphan Drug Act (ODA) came into effect in 1983 as a solution to meet an unmet need in the industry. Its success is often projected as an example of how innovative regulatory frameworks can be used ...

We recently published 13 Best ADR Stocks to Invest In. GSK plc (NYSE:GSK) is one of the best ADR stocks. GSK plc (NYSE:GSK) is one of the largest pharmaceutical companies in the world. The firm scored ...

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 ...

Last month, the Food and Drug Administration took the unusual step of admitting it had made a mistake 25 years ago when it granted orphan drug status to an opioid addiction treatment called ...

The Orphan Drug Act of 1983 was passed to promote the development of rare disease treatments. To qualify for incentives under the act, sponsors must first receive “orphan drug designation” from the ...

If you've ever thought about investing in a company that develops medicines for rare diseases, you've probably recognized the market-creating impact of orphan drug designations. Orphan drugs are ...