‘Life-Changing’ Hemophilia Treatment Could Be Priciest Ever: $3M Per Patient
Read about the biggest pharmaceutical developments and pricing stories from the past week in KHN's Prescription Drug Watch roundup.
NPR:
Gene Therapy Drug For Hemophilia May Be Priced As High As $3 Million Per Patient
One of the biggest questions is the possible cost. BioMarin Pharmaceutical Inc. of San Rafael, Calif., the company that developed the gene therapy, says the treatment could cost as much as $3 million per patient, which would make it the most expensive drug ever approved. "It's just outrageous," says Peter Bach, who studies drug prices at Memorial Sloan Kettering Cancer Center in New York. (Stein, 7/20)
Stat:
Acadia Fails In Attempt To Use Antipsychotic To Treat Patients With Depression
Acadia Pharmaceuticals reported negative results Monday from a pair of late-stage clinical trials seeking to expand the use of its antipsychotic medicine Nuplazid to patients with major depressive disorder. The two identically designed Phase 3 clinical trials involved 300 patients who hadn’t responded well to currently approved depression treatments. In both studies, Nuplazid failed to demonstrate an anti-depressive benefit compared to a placebo when given to patients alongside their current medicines. (Feuerstein, 7/20)
Reuters:
Britain Secures 90 Million Possible COVID-19 Vaccine Doses From Pfizer/BioNTech, Valneva
Britain has signed deals to secure 90 million doses of two possible COVID-19 vaccines from an alliance of Pfizer Inc and BioNTech, and French group Valneva, the business ministry said on Monday. Britain secured 30 million doses of the experimental BioNTech/Pfizer vaccine, and a deal in principle for 60 million doses of the Valneva vaccine, with an option of 40 million more doses if it was proven to be safe, effective and suitable, the ministry said. With no working vaccine against COVID-19 yet developed, Britain now has three different types of vaccine under order and a total of 230 million doses potentially available. (Smout, 7/20)
Politico:
An Indian Drug Mogul Says Americans Will Pay Too Much For The Covid Vaccine—And Wants To Change That
Adar Poonawalla may be the most important figure in the global vaccine race who isn’t working in a laboratory: The Indian vaccine entrepreneur plans to save the world from coronavirus – and then radically remake the international pharma landscape. Drug companies are sounding alarms. The globe-trotting, deal-making son of Dr. Cyrus Poonawalla – an Indian billionaire who founded the Serum Institute of India on his horse farm 54 years ago – is one of the breakout figures of the Covid fight, strategizing from the Indian city of Pune to bring the same low-cost efficiency with which he makes 1.5 billion vaccine doses a year for the developing world into mass-producing a Covid-19 vaccine for the entire world. (Wheaton, 7/19)
The New York Times:
Inside Johnson & Johnson’s Nonstop Hunt For A Coronavirus Vaccine
Each workday morning in March, Noe Mercado drove through the desolate streets of Boston to a tall glass building on Blackfan Circle, in the heart of the city’s biotech hub. Most residents had gone into hiding from the coronavirus, but Mr. Mercado had an essential job: searching for a vaccine against this new, devastating pathogen. Parking in the underground lot, he put on a mask and rode the empty elevator to the tenth floor, joining a skeleton crew at the Center for Virology and Vaccine Research at Beth Israel Deaconess Medical Center. Day after day, Mr. Mercado sat at his lab bench, searching for signs of the virus in nasal swabs taken from dozens of monkeys. (Zimmer, 7/17)
