Although the World Health Organization (WHO) acknowledged COVID-19 is spreading, they say it has not yet reached pandemic disease status. The statement did little to comfort the nerves of investors Feb. 24 as they exited global markets big time in the wake of an eruption of new infections in Italy, Iran and South Korea.
Valrox (valoctocogene roxaparvovec) from San Rafael Calif-based Biomarin Pharmaceutical Inc. moved one step closer to entering the U.S. market, with the company reporting that that the FDA had accepted for priority review the BLA for its investigational AAV5 gene therapy for adults with hemophilia A.
The BioWorld Cancer index, which includes 21 representative companies developing therapies targeting various cancers, entered the new year on a high note, after posting a 22.5% gain for the year. Unfortunately, the group hit a speed bump and the index took a beating in January, dropping almost 9% as a result.
Millions of people are affected by blood disorders, and the prevalence is expected to grow as our population ages. It is not surprising that, according to the American Society of Hematology, the FDA approved several new therapies – or new indications for previously approved therapies – in 2019 for people living with non-malignant blood disorders. Those included two disease-modifying treatments for sickle cell disease and the first anticoagulant for venous thromboembolism management in children.
Investors in small and midsized biopharma companies were certainly rewarded in 2019, with group members in the BioWorld Drug Developers index on a tear. The price-weighted index returned 40% in value thanks to a steady flow of positive regulatory and clinical trial results from the companies throughout the year. However, investors may be less impressed with the start they have made this year, with the index dipping 8.4% in January.
Biotech investors had every reason to feel bullish heading into the new decade. The sector had turned around in 2019 and was riding a wave of a very strong fourth-quarter performance, with the BioWorld Biopharmaceutical Index closing up 14% for the year after being underwater from April through to September. Unfortunately, those great expectations were quickly erased during J.P. Morgan Healthcare conference week (Jan. 10 – Jan. 17), which turned out to be a very low-key affair absent of any blockbuster M&A revelations. As a result, confidence has now given way to concerns about the prospects for biopharmaceutical companies going forward, particularly as unfavorable political rhetoric on drug pricing will certainly be dialed up during this election year.
