The acceptance by the FDA of Prevail Therapeutics Inc.’s IND for the one-time, fast-tracked gene therapy PR-006 provided hope for 50,000 to 60,000 people in the U.S beset by frontotemporal dementia with the GRN mutation (FTD-GRN), and the New York-based company is moving ahead with a phase I/II experiment called Proclaim.
With a coronavirus task force briefing unfolding at the White House late on April 17, an FDA eager to show its ongoing commitment to tackling other disease amid the pandemic, granted accelerated approval to Incyte Corp.'s Pemazyre (pemigatinib), the first treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma.
Looking ahead to COVID-19 strategies, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), said vaccine studies might be done not in clinics but in the streets. “By definition, we’re probably not going to be able to vaccinate everyone simultaneously,” he noted.
As expected – and well ahead of the Aug. 20 PDUFA date – Bothell, Wash-based Seattle Genetics Inc. (Seagen) won FDA clearance for the oral small-molecule breast cancer therapy tucatinib, a tyrosine kinase inhibitor branded Tukysa.
HONG KONG – South Korean biopharmaceutical company Kolon Tissuegene Inc. got a new lease on life as the U.S. FDA lifted the hold on the phase III trial for its lead candidate, Invossa-K (Invossa), for the treatment of osteoarthritis.
The FDA approved Urogen Pharma Ltd.’s mitomycin gel, an orphan drug branded Jelmyto, on April 15, offering patients the first non-surgical option for low-grade upper tract urothelial cancer (LG-UTUC) and granting the Princeton, N.J.-based company with its first marketed product.
An April 15 U.S. FDA stakeholder call revisited several themes of interest in connection with diagnostics for the COVID-19 pandemic. However, Tim Stenzel, director of the agency’s Office of In Vitro Diagnostics and Radiological Health, said that while the agency has not yet authorized a home sample collection kit, “we do think it’s going to happen very soon.”
Nearly 40 years on, the generic drug market is often lauded as an American success story. But a closer inspection reveals such back-patting ignores the potential for serious public health risks caused by ongoing shortages in the generic drug supply. It also ignores quality issues and lingering physician and patient doubts about generics, especially those made in other countries.
The age of molecular testing for the COVID-19 pandemic is still with us, but the emphasis in the months ahead will be on serological testing as a quicker, more useful mass testing alternative. However, test developers have a number of hurdles to overcome in devising these serological tests, including that antibodies for the virus’s antigens emerge at different times in the disease cycle, just one of several challenges that have to be met in the effort to bring the SARS-CoV-2 virus to heel.
When it comes to guidance on interchangeables, the Pharmaceutical Research and Manufacturers of America (PhRMA) has some guidance for the FDA: Be more definite.
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