Getting to Market
The U.S. Food and Drug Administration (FDA) will have some tough decisions to make. BioPharma Dive listed clearing an experimental Alzheimer’s drug from Biogen, approving a coronavirus vaccine from AstraZeneca that has been the subject of an unusual public dispute between the drugmaker and its independent trial review board, changing the standard of care for a rare genetic disease and clearing a new vaccine for one of the world’s most common infections as top priorities. (https://www.biopharmadive.com/news/5-fda-approvals-second-quarter-2021/597474/?utm_source=Sailthru&utm_medium=email&utm_campaign=Issue:%202021-03-29%20BioPharma%20Dive%20%5Bissue:33278%5D&utm_term=BioPharma%20Dive)
According to the article, “Any rejection could add to an impression that the FDA is becoming stricter of late, after a series of surprising regulatory knockbacks and an industry-wide evaluation of cancer immunotherapy approvals. Clearance of Biogen’s drug, meanwhile, would have the opposite effect.” Here are five drugs, listed in order of the date by which the FDA is expected to make a decision on approval.
Acadia Pharmaceuticals’ pimavanserin for dementia-related psychosis
Pimavanserin, the only drug on the market from Acadia Pharmaceuticals, has been sold under the brand name Nuplazid to treat Parkinson’s disease psychosis. Last year, the company asked for an approval in dementia-related psychosis. The FDA decision date is slated for April 3.
Acadia executives expected a good outcome until regulators found “deficiencies” in the company’s application. FDA feedback appears to have ruled out an April approval and reduced the chance of an eventual one.
AstraZeneca and Oxford’s coronavirus vaccine
While three coronavirus vaccines available for use in the U.S. got through the FDA without drama, the one from AstraZeneca has encountered communication missteps and safety setbacks. Positive but confusing data left it unclear how to best use the shot or whether it worked as well in the elderly. A trial stoppage of almost two months delayed a larger trial in the U.S. and South America, and then rare, abnormal blood clots have occurred during the shot’s roll-out in Europe.
While AstraZeneca recently reported that the shot was safe and strongly effective at preventing COVID-19 in the U.S. study, the board of independent experts overseeing the trial publicly disputed the initial results.
Sanofi’s avalglucosidase alfa for Pompe disease
Pompe disease involves a genetic mutation that stops people’s bodies from producing a key enzyme that breaks down glycogen. The main treatment for Pompe has been enzyme replacement therapy, called Lumizyme, that Sanofi acquired when it bought Genzyme. Sanofi wants to set a new treatment standard by winning approval of a successor therapy called avalglucosidase alfa to deliver more of the enzyme into cells, improving potency. In a Phase 3 study, avalglucosidase alfa matched Lumizyme as measured by improvements in respiratory function, but the “clinical relevance” of the difference between how the two drugs work has not been established yet.
Still, approval of Sanofi’s new drug could raise the bar for a number of other treatments in development. A decision is expected by May 18.
Biogen’s aducanumab for Alzheimer’s disease
Alzheimer’s disease remains one of the most elusive areas of drug development. The FDA should decide by June 7 whether to approve Biogen’s aducanumab, which would be the first marketed drug to slow the progression of Alzheimer’s.
However, FDA statisticians, as well as external advisors, have criticized the aducanumab data and the ways Biogen analyzed it. In a November meeting, those advisors voted almost unanimously against the drug. Still, the FDA is under pressure to get more Alzheimer’s drugs to patients, and the agency’s clinical staff members have been supportive of Biogen’s application. When regulators decided to extend their review of aducanumab, it may have been a sign that more data could make the difference.
Pfizer’s 20-valent pneumococcal vaccine
Both Pfizer and Merck & Co. have approved vaccines to prevent pneumococcal disease. Neither protects against key emerging strains of the infectious bacteria.
Pfizer’s vaccine stimulates immunity to 20 different strains of disease-causing bacteria. It protects against the same 13 serotypes that the current standard, Prevnar 13, does, plus seven more that have been associated with higher death rates, antibiotic resistance or meningitis. By June, the FDA will decide whether to approve Pfizer’s 20-valent shot for people 18 years and older. Prevnar can be used in children and infants as young as six weeks, but Pfizer aims to eventually treat that group as well, having published Phase 2 data for the new vaccine in infants and children.
Merck expects a decision July 18 for its 15-valent pneumococcal vaccine called V114 in adults 18 and older. V114 would replace the company’s older 23-valent shot called Pneumovax 23 and protect against key strains that are helping drive disease.
