A year after finding flaws in the biotech’s application, the regulator cleared a treatment some analysts view as a blockbuster-to-be, sending shares up by as much as 40%.

The biotech startup, which licensed two programs from Novartis, is part of a crowded field of drugmakers trying to develop new hepatitis B treatments. 

Approved Wednesday for severe beta thalassemia, Zynteglo will test insurers’ willingness to pay for expensive one-time treatments. Its market launch is likely to be watched carefully by other gene therapy developers.

The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with gene editing and RNA medicines.

Geoffrey Porges, who has spent nearly two decades researching biotechs at SVB Securities and AllianceBernstein, will now oversee and seek to grow the drug discovery specialist's network of partnerships.

The federal government will begin the transition as early as this fall, clearing the way for drugmakers to control sales and distribution of their shots and therapeutics.  

The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a very high cost of $2.8 million in the U.S. 

The French drugmaker’s shares fell by more than 6% after the once promising treatment, called amcenestrant, failed its second major trial this year.

The COVID-19 vaccine maker has appointed James Mock, currently PerkinElmer’s CFO, as its next finance head, three months after ousting Jorge Gomez from the role. 

Trial results position the company to seek approval for indolent systemic mastocytosis, which executives have painted as a “multibillion dollar” market opportunity. Even so, shares fell by double digits in trading Wednesday.

The company has agreed to spend at least $280 million buying back rights to the drug, called Trodelvy, which were sold to Everest Medicines in 2019.

The biotech plans to start Phase 2 studies for a drug it believes may more effectively hit its target than Bristol Myers’ deucravacitinib, a skin disease treatment regulators could approve next month.