“It’s a rare window into seeing how the FDA makes decisions because the proceedings are open unlike many of the other decisions they make,” Audrey Zhang, a medical student at the New York University School of Medicine, said on why she decided to embark on crunching the numbers from 376 votes by FDA advisory committee meetings from 2008 to 2015.
Every year at the Cleveland Clinic Medical Innovation Summit, the Top 10 list of technologies that are predicted to come to the market and change patient care is revealed. Last year's October event saw RNA-based therapies take the No. 10 spot.
Despite the ups and downs of the general markets and a U.S. government shutdown at the beginning of the year that contributed to no biopharma companies graduating to the public ranks in January, the enthusiasm for biopharma IPOs has remained steady since then. In fact, a flurry of IPO listings on U.S. stock exchanges last month helped bring the total of those offerings to 30 at the halfway point in the year.
Drugs for rare diseases now account for 31% of R&D pipelines, up from 18% in 2010 and just 11% in 2005, according to a report from the Tufts Center for the Study of Drug Development. That's currently nearly 3,500 drugs in development, more than double the 1,530 in 2010.
Although the public biopharmaceutical sector set a fast pace at the beginning of the year, it has been all downhill since February, with the BioWorld Biopharmaceutical index losing 16% of its value. However, a strong performance in June, with the group gaining 7.6%, helped the sector marginally push into positive territory year-to-date (YTD). At the halfway point of the year, the index is trailing the general market by a wide margin, with the Dow Jones Industrial Average trading up 14% and the Nasdaq Composite index up 20%.
DUBLIN – European biotechnology firms engaged in drug discovery and development raised an aggregate $3.172 billion in equity investment during the first half of 2019, down 19% on the same period last year. Unless there is a substantial pickup in the third and fourth quarters, the sector's record-breaking 2018 total of $7.715 billion looks to be out of reach.
In late May, Novartis AG's Avexis Inc. unit gained FDA approval for Zolgensma (onasemnogene neparvovec) to treat spinal muscular atrophy, and other companies are looking to follow suit developing drugs to treat a variety of diseases of the central nervous system (CNS).
It has been acknowledged by governments around the world that there are simply not enough antibiotics currently in the product pipeline to meet the predicted requirements of their health care systems. Not good news considering that antibiotic resistance continues to be a major societal concern, with the CDC estimating that in the U.S. alone at least 2 million people contract an antibiotic-resistant infection, and at least 23,000 people die annually as a result.