Government
Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson launches an investigation into recent rare disease drug rejections; and Roche and Zealand’s amylin analog fails to match investor expectations—and Eli Lilly’s rival candidate—in a mid-stage trial.
Some biotechs that had seen regulatory setbacks under Center for Biologics Evaluation and Research director Vinay Prasad experienced stock bumps Monday morning. Under Prasad’s leadership, the rare disease space has suffered a series of controversial rejections.
TrumpRX and DTC sales may expand prescription drug access, but they will not solve the affordability crisis by themselves.
Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg and is a fellow skeptic of the use of selective serotonin reuptake inhibitors during pregnancy.
UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.
One of the two new members of the CDC’s Advisory Committee on Immunization Practices questioned the safety of COVID-19 vaccines before the Texas Senate in 2021.
The CDC’s changes threaten to cut vaccine sales for makers including Pfizer, Moderna, Merck and more, but a legal expert suspects affected manufacturers will stay on the sidelines rather than back a push to declare the revised schedule unlawful.
This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite these treatments.
In his State of the Union address on Tuesday evening, President Donald Trump urged Republican leaders to pass legislation cementing his new drug pricing initiatives as he touted his efforts to lower healthcare costs for Americans.
The rescheduling of the Advisory Committee on Immunization Practices comes amid fresh leadership upheaval at the CDC as NIH director Jay Bhattacharya replaces Jim O’Neill as acting head of the agency.
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