It’s of­fi­cial: blue­bird bio has scored its first drug ap­proval with an EU mar­ket­ing au­tho­riza­tion for its gene ther­a­py for trans­fu­sion-de­pen­dent β-tha­lassemia.

Zyn­te­glo is the new name for a treat­ment once known as Lenti­Glo­bin, a process that takes CD34+ cells from a pa­tient’s hematopoi­et­ic stem cells, in­serts func­tion­al copies of the βA-T87Q-glo­bin gene, then re­in­fus­es the mod­i­fied HSCs. Pa­tients are giv­en chemother­a­py to prime their bone mar­rows be­fore the re­in­fu­sion.

It’s a one-shot ther­a­py de­signed to re­place a life­time of trans­fu­sions and med­ica­tion, in­di­cat­ed for pa­tients 12 years or old­er. The ap­proval fol­lows an EMA rec­om­men­da­tion in March.

The Cam­bridge, MA-based biotech plans to pro­vide an up­date on the roll out plans and pric­ing by the end of next week, a spokesper­son con­firmed.

No mat­ter the fig­ure, the stick­er price is like­ly to trig­ger a fresh wave of de­bate on pric­ing of sup­pos­ed­ly once-and-done ther­a­pies while No­var­tis’ de­ci­sion to charge $2.1 mil­lion for its spinal mus­cu­lar at­ro­phy treat­ment still re­ver­ber­ates.

While blue­bird has pro­duced com­pelling re­sults — dra­mat­i­cal­ly re­duc­ing the need for blood trans­fu­sions in a 22-pa­tient study and elim­i­nat­ing it in most of the pa­tients with a less se­vere form of the dis­ease — Zyn­te­glo has fall­en short of a cure for more se­vere pa­tients. Phase III tri­als are on­go­ing.

Chief ex­ec­u­tive Nick Leschly has pre­vi­ous­ly pro­posed a plan to pay for the ther­a­py by in­stall­ment and stat­ed that Zyn­te­glo will be priced low­er than its “in­trin­sic val­ue” — co­in­ci­den­tal­ly es­ti­mat­ed at $2.1 mil­lion

SVB Leerink an­a­lysts have pre­vi­ous­ly mod­eled the launch price of Lenti­Glo­bin as $1.2 mil­lion in the US and $0.9 mil­lion in the EU.

Im­age: Nick Leschly. In­ter­na­tion­al So­ci­ety for Phar­ma­ceu­ti­cal En­gi­neer­ing

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.