If there was an award for the most accurate nomenclature in clinical development, window of opportunity (WOO) studies would certainly be in the running since they provide just that: an opportunity to test a drug during the window between diagnosis and excising of the primary tumor.

It was a mixed bag for Sangamo Therapeutics Inc. at the 2018 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Athens, Greece, on Wednesday where the company presented data from its phase I/II Champions trial testing SB-913 in patients with mucopolysaccharidosis (MPS) type II.

With multiple recent approvals of biologics to treat psoriasis Cosentyx (secukinumab, Novartis AG), Ilumya (tildrakizumab-asmn, Sun Pharmaceutical Industries Ltd.), Taltz (ixekizumab, Eli Lilly and Co.) and others companies are turning to topical treatments to capture the estimated 80 percent to 90 percent of psoriasis patients with mild to moderate disease where cheaper topical medications are more appropriate.

Takhzyro's (lanadelumab-flyo) breakthrough designation was only good for a one-business-day advantage in gaining FDA approval ahead of its Prescription Drug User Fee Act action date on Sunday.

Ask the average drug company CEO about rebates given to pharmacy benefit managers (PBMs), and you'll get an earful about how they aren't good for the system. But companies have continued to play along because of a lack of other options.

For many drugs with targets in the central nervous system, it may not be the neurology target that keeps the drugs from succeeding in clinical trials, but the ability of the drug to get past the blood-brain barrier in high enough concentration to be effective.

It's been more than two decades since Doxil (doxorubicin, Johnson & Johnson), a liposome-based chemotherapy, was approved by the FDA. During that time, the field has evolved from simply protecting other tissue from highly potent drugs until they get to the site of the disease to increasing targeting and bioavailability.

It's been more than two decades since Doxil (doxorubicin, Johnson & Johnson), a liposome-based chemotherapy, was approved by the FDA. During that time, the field has evolved from simply protecting other tissue from highly potent drugs until they get to the site of the disease to increasing targeting and bioavailability.

The quest to get the first drug approved to treat fragile X is heating up with three companies – Tetra Discovery Partners Inc., Zynerba Pharmaceuticals Inc. and Ovid Therapeutics Inc. – starting clinical trials this month.