Since the FDA's breakthrough therapy designation came into existence as part of the FDA Safety and Innovation Act in July 2012, the agency has granted the designation to 145 drugs.
Genomic sequencing offers great hope for patients to treat their disease in the most specific way possible through precision medicine. But the new frontier in medicine isn't very helpful for patients with diseases that don't have known links to genetic mutations.
Small companies often avoid the heart drug market, staying clear of the waters typically traversed by larger drugmakers who are more able to handle the risk associated with the larger and longer clinical trials required.
Earlier this month, Shire plc gained FDA approval for Xiidra (lifitegrast), the first drug approved for dry eye disease since Allergan Inc.'s Restasis (cyclosporine) was approved 14 years ago.
In the first quarter of 2016, private U.S. biopharmas tracked by BioWorld Snapshots raised about $380 million less than the same quarter in 2015, but the year-over-year decline was easy to shrug off because the first quarter of 2015 contained a monster $450 million series C round by Moderna Therapeutics Inc.
Diabetes drugs have come a long way from meta-analyses from Steven Nissen, chairman of cardiovascular medicine at the Cleveland Clinic, and others suggesting that Avandia (rosiglitazone, Glaxosmithkline plc) was increasing the risk of cardiovascular (CV) problems.
Gene therapy has come a long way in the decades since it was first conceived, with eye diseases being one of the major focuses for companies big and small alike.
About 30 million Americans, roughly 10 percent of the population, has one of the 7,000 known rare diseases, defined as affecting fewer than 200,000 people in the U.S. And rare diseases unfortunately affect children disproportionately, making up half of all cases globally.
Having large databases can be helpful in making discoveries not obvious in smaller datasets, but getting the data into a compatible format and then actually using the “big data” in a meaningful manner can be challenging.
