Regulation

EC approves ZYNTEGLO gene therapy for transfusion-dependent β-thalassemia

bluebird bio has secured conditional marketing authorisation from the European Commission (EC) for its one-time gene therapy ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene) for the treatment of transfusion-dependent β-thalassemia (TDT).

FDA approves new treatment for hospital-acquired and ventilator-associated bacterial pneumonia

The U.S. Food and Drug Administration today approved a new indication for the previously FDA-approved drug, Zerbaxa (ceftolozane and tazobactam) for the treatment of hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) in patients 18 years and older.

Vertex to submit VX-445, Tezacaftor and Ivacaftor regimen for global regulatory approvals in cystic fibrosis

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has selected the triple combination of the next-generation corrector VX-445 (elexacaftor), tezacaftor and ivacaftor to submit for potential global regulatory approvals for people ages 12 and older with cystic fibrosis (CF).

Epizyme submits NDA to US FDA for tazemetostat to treat epithelioid sarcoma

Epizyme has submitted a new drug application (NDA) to the US Food and Drug Administration (FDA) for accelerated approval of tazemetostat for the treatment of patients with metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery.

Merz gets European approval for Xeomin to treat chronic sialorrhea in adults

German neurotoxin firm Merz has secured European approval for its Xeomin (incobotulinumtoxinA) for the symptomatic treatment of chronic sialorrhea (drooling) due to neurological disorders in adult patients.

Santhera seeks EMA approval for idebenone in Duchenne muscular dystrophy

Santhera Pharmaceuticals has filed a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the approval of Puldysa (idebenone) for the treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD).

Celgene gets FDA nod for blood cancer drug combo Revlimid plus rituximab

Celgene has secured approval from the US Food and Drug Administration (FDA) for its Revlimid (lenalidomide) in combination with a rituximab product (R²) to treat adult patients with previously treated follicular lymphoma (FL) or marginal zone lymphoma (MZL).

Teva reaches agreement with Oklahoma to resolve state’s claims

Teva Pharmaceuticals USA and related affiliates of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), and the state of Oklahoma, have entered into an agreement for a one-time payment of $85 million to the state.

Novartis gets FDA nod for breast cancer drug combo Piqray plus fulvestrant

Novartis has secured approval from the US Food and Drug Administration (FDA) for its breast cancer drug combo Piqray (alpelisib, formerly BYL719) plus fulvestrant.